Materiały źródłowe

• #1 Prophylaxis in haemophilia should be life-long

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3320774/

  Prophylaxis in haemophilia should be life-long. […] Although prophylaxis is now considered the gold standard for the treatment of severe haemophilia in childhood and adolescence, its use in adulthood is more controversial but here I argue that prophylaxis should be the gold standard in haemophilia treatment for life. […] The evidence that prophylaxis is effective is less strong in adults than it is in children but it is nevertheless convincing. […] For the majority of patients with standard severe haemophilia, however, adult prophylaxis should be the standard of care in the future as it will prevent bleeds, arthropathy and improve quality of life. […] Ultimately, as the authors of the editorial to accompany Manco-Johnsons paper in the New England Journal of Medicine state, „The dilemma in managing haemophilia is not whether to use prophylaxis or episodic treatment but how to manage prophylaxis such that the optimal, most cost effective treatment is provided”. […] I believe the statement applies equally well to the adult situation and for the majority of patients with severe haemophilia prophylactic treatment for life should be the new gold standard of care.

• #2 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  Starting from the clinical observations that moderate haemophiliacs experienced only few bleeding episodes and rarely developed significant joint deterioration (haemophilic arthropathy), and the pioneer experience in Sweden, prophylaxis (i. e. the regular and long-term administration of clotting factor concentrate in order to prevent bleeding) has been practiced for more than forty years in severe haemophilia and is currently recommended as the first choice of treatment by the World Health Organisation and World Federation of Hemophilia and by many national medical/scientific organizations. […] Observational studies clearly established the superiority of prophylaxis over on-demand treatment in reducing the risk of arthropathy, also showing that starting prophylaxis earlier in life and after very few joint bleeds was associated with better joint outcomes, and led to the current definitions of primary (started before the age of 2 yrs and after no more than one joint bleed) and secondary prophylaxis.

• #3 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  In patients with severe haemophilia, prophylaxis, i.e. the infusion of factor concentrate replacement treatment in order to prevent bleeding episodes, has been used for more than 40 years in Northern Europe and more recently in other European countries and in Northern America, becoming the first-choice treatment recommended by the World Health Organisation (WHO) and the World Federation of Haemophilia since 1994. […] The main objective of the prophylactic replacement treatment since the Swedish pioneering regimens was (and remains) to minimise the number of joint bleeds since an early age by converting the severe form of haemophilia to a milder form. […] Preventing or reducing the clinical impact of haemophilic arthropathy by prophylaxis means enabling normal life and psychosocial development for haemophilic children, including the possibility of physical activities, regular school attendance and, consequently, social and work opportunities.

• #4 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  Starting from the clinical observations that moderate haemophiliacs experienced only few bleeding episodes and rarely developed significant joint deterioration (haemophilic arthropathy), and the pioneer experience in Sweden, prophylaxis (i. e. the regular and long-term administration of clotting factor concentrate in order to prevent bleeding) has been practiced for more than forty years in severe haemophilia and is currently recommended as the first choice of treatment by the World Health Organisation and World Federation of Hemophilia and by many national medical/scientific organizations. […] Observational studies clearly established the superiority of prophylaxis over on-demand treatment in reducing the risk of arthropathy, also showing that starting prophylaxis earlier in life and after very few joint bleeds was associated with better joint outcomes, and led to the current definitions of primary (started before the age of 2 yrs and after no more than one joint bleed) and secondary prophylaxis.

• #5 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  In patients with severe haemophilia, prophylaxis, i.e. the infusion of factor concentrate replacement treatment in order to prevent bleeding episodes, has been used for more than 40 years in Northern Europe and more recently in other European countries and in Northern America, becoming the first-choice treatment recommended by the World Health Organisation (WHO) and the World Federation of Haemophilia since 1994. […] The main objective of the prophylactic replacement treatment since the Swedish pioneering regimens was (and remains) to minimise the number of joint bleeds since an early age by converting the severe form of haemophilia to a milder form. […] Preventing or reducing the clinical impact of haemophilic arthropathy by prophylaxis means enabling normal life and psychosocial development for haemophilic children, including the possibility of physical activities, regular school attendance and, consequently, social and work opportunities.

• #6 Hemophilia A & B Prophylaxis – Guidelines Side-By-Side – Guideline Central

  https://www.guidelinecentral.com/insights/hemophilia-side-by-side/

  Hemophilia A and B, the most common severe hereditary bleeding disorders, arise from deficiencies in factor VIII and factor IX, respectively. Apart from treating acute bleeding, prophylaxis is a key strategy in hemophilia management. Prophylactic treatment significantly reduces the frequency of hemarthroses, thereby mitigating hemophilic arthropathy and the need for corrective joint surgeries. It also lowers the incidence of cerebral and muscle bleeds, decreases hospitalizations, and enhances patients quality of life by reducing time off work and less frequent monitoring. […] For patients with hemophilia A or B with a severe phenotype: Strongly recommend patients be on prophylaxis sufficient to prevent bleeds at all times in an individualized approach. […] For patients with severe phenotype hemophilia A or B, especially children: Recommend regular long-term prophylaxis as the standard of care to prevent hemarthrosis and other spontaneous and breakthrough bleeding, maintain musculoskeletal health, and promote quality of life.

• #7 What You Need to Know About Prophylaxis | HemAware

  https://hemaware.org/bleeding-disorders-z/what-is-prophylaxis

  This preventive treatment can help people with hemophilia avoid joint damage. […] For children with severe hemophilia, prophylaxis is the standard of care. Studies show that starting children with severe hemophilia on prophylaxis early (before age 3 and prior to the second joint bleed) is the most effective way to preserve healthy joint function over their lives, and to prevent other complications. […] Today, many doctors also recommend prophylaxis for adults with severe hemophilia. […] When scheduled factor infusions begin before bleeding episodes have occurred, this is called primary prophylaxis. Beginning prophylaxis after two joint bleeds but before any sign of joint damage is called secondary prophylaxis. The goal for both is to prevent spontaneous joint bleeds and the development of joint disease. […] Tertiary prophylaxis refers to regularly scheduled factor infusions that occur after someone has had several joint bleeds and has evidence of some joint damage. The purpose of tertiary prophylaxis is to allow the joint to heal and prevent more bleeding or further joint damage.

• #8 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  Starting from the clinical observations that moderate haemophiliacs experienced only few bleeding episodes and rarely developed significant joint deterioration (haemophilic arthropathy), and the pioneer experience in Sweden, prophylaxis (i. e. the regular and long-term administration of clotting factor concentrate in order to prevent bleeding) has been practiced for more than forty years in severe haemophilia and is currently recommended as the first choice of treatment by the World Health Organisation and World Federation of Hemophilia and by many national medical/scientific organizations. […] Observational studies clearly established the superiority of prophylaxis over on-demand treatment in reducing the risk of arthropathy, also showing that starting prophylaxis earlier in life and after very few joint bleeds was associated with better joint outcomes, and led to the current definitions of primary (started before the age of 2 yrs and after no more than one joint bleed) and secondary prophylaxis.

• #9 What You Need to Know About Prophylaxis | HemAware

  https://hemaware.org/bleeding-disorders-z/what-is-prophylaxis

  This preventive treatment can help people with hemophilia avoid joint damage. […] For children with severe hemophilia, prophylaxis is the standard of care. Studies show that starting children with severe hemophilia on prophylaxis early (before age 3 and prior to the second joint bleed) is the most effective way to preserve healthy joint function over their lives, and to prevent other complications. […] Today, many doctors also recommend prophylaxis for adults with severe hemophilia. […] When scheduled factor infusions begin before bleeding episodes have occurred, this is called primary prophylaxis. Beginning prophylaxis after two joint bleeds but before any sign of joint damage is called secondary prophylaxis. The goal for both is to prevent spontaneous joint bleeds and the development of joint disease. […] Tertiary prophylaxis refers to regularly scheduled factor infusions that occur after someone has had several joint bleeds and has evidence of some joint damage. The purpose of tertiary prophylaxis is to allow the joint to heal and prevent more bleeding or further joint damage.

• #10 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  Starting from the clinical observations that moderate haemophiliacs experienced only few bleeding episodes and rarely developed significant joint deterioration (haemophilic arthropathy), and the pioneer experience in Sweden, prophylaxis (i. e. the regular and long-term administration of clotting factor concentrate in order to prevent bleeding) has been practiced for more than forty years in severe haemophilia and is currently recommended as the first choice of treatment by the World Health Organisation and World Federation of Hemophilia and by many national medical/scientific organizations. […] Observational studies clearly established the superiority of prophylaxis over on-demand treatment in reducing the risk of arthropathy, also showing that starting prophylaxis earlier in life and after very few joint bleeds was associated with better joint outcomes, and led to the current definitions of primary (started before the age of 2 yrs and after no more than one joint bleed) and secondary prophylaxis.

• #11 Hemophilia A and B Prophylaxis Recommendations | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-267-masac-recommendation-concerning-prophylaxis-for-hemophilia-a-and-b-with-and-without-inhibitors

  In view of the demonstrated benefits of prophylaxis begun at a young age in persons with hemophilia A or B (PwH), MASAC recommends that prophylaxis be considered standard of care therapy for individuals with severe hemophilia A or B (factor VIII or factor IX 1%) including those with inhibitors. Prophylactic therapy may also be considered for PwH with moderate and mild hemophilia with a severe phenotype. Prophylactic therapy should be instituted early (prior to the onset of frequent bleeding). […] Prophylaxis should be initiated at an early age, ideally before age 3 years and prior to the second joint bleed; prophylaxis may be considered within the first six months of life to reduce occurrence of intracranial hemorrhage. […] The goals for emicizumab prophylaxis remain like that of factor prophylaxis in that a regular administration of a prophylactic hemostatic agent should be considered as early as feasible to prevent the long-term complications of musculoskeletal bleeding and allow the patient to lead a physically active life approaching the quality of life of an unaffected individual.

• #12 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  Long-term clinical outcome was more favourable in patients starting prophylaxis before the age of 4 (no arthropathy in 50% of patients vs. 21% in those starting between 3 and 7 years old), with an estimated 8% higher Pettersson score per year of postponed prophylaxis from birth, and the best results (70% of patients with score 0) found in patients with fewer than three bleeds before the start of prophylaxis. […] The relative rarity of the disease and the difficulty in performing randomised controlled studies (especially in countries in which consolidated experience on prophylaxis raises ethical considerations on possible randomisation of children to on-demand treatment) until recently, led the Cochrane reviewers in 2005 to identify only four small randomised cross-over trials, comparing a prophylactic regimen with placebo or two different prophylactic regimens.

• #13 Hemophilia A (Factor VIII Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779322-treatment

  In most developed countries with access to recombinant product, prophylaxis is primary (ie, therapy is started in patients as young as 1 y and continues into adolescence). A cost-benefit analysis indicates that this approach reduces overall factor use and significantly reduces morbidity. […] For prophylaxis, dosing is designed to maintain trough levels of 2% or higher. This usually requires the administration of FVIII three times per week. Individualized therapy (ie, tailored prophylaxis) has been also used with success; the best approach has yet to be determined. Monoclonal antibodies (ie, emicizumab, marstacimab) may also be used for prophylaxis. […] Most of the care for children with severe hemophilia now takes place at home, in the community, and at school, allowing these children to participate in normal activities that are otherwise impossible. This approach resulted from the development of prophylactic regimens of factor concentrate infusions that are administered at home, usually by a parent.

• #14 Hemophilia B (Factor IX Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779434-treatment

  A cost-benefit analysis indicates that this approach reduces overall factor use and significantly reduces morbidity. […] Dosing is designed to maintain trough levels greater than 2%. […] With the development of FIX preparations that have extended half-lives, dosing for routine prophylaxis may be as infrequent as every 10 days. […] An option for prophylaxis is marstacimab, a monoclonal antibody directed against tissue factor pathway inhibitor, which was approved by the US Food and Drug Administration (FDA) in October 2024. […] Another such option is fitusiran, an antithrombin-directed small interfering ribonucleic acid (RNA), which received FDA approval in March 2025. […] Gene therapy offers the potential for a definitive cure and has entered clinical practice, with FDA approval of the first product in 2022.

• #15 Individualized prophylaxis for optimizing hemophilia care: can we apply this to both developed and developing nations? | Thrombosis Journal | Full Text

  https://thrombosisjournal.biomedcentral.com/articles/10.1186/s12959-016-0096-y

  Prophylaxis is considered optimal care for hemophilia patients to prevent bleeding and to preserve joint function thereby improving quality of life (QoL). […] Prophylaxis can be further individualized to improve outcomes and cost effectiveness. […] Prophylaxis in economically constrained nations is limited by the ill-affordability of clotting factor concentrates. […] However, at least 5 studies on children and adults from Thailand, China and India have shown superiority of low dose (~510 IU kg1 2-3 per week) prophylaxis over episodic treatment in terms of bleed reduction, and quality of life, with improved physical activity, independent functioning, school attendance and community participation. […] Prophylaxis must be individualized to include affordability as a major determinant in order to optimize low-dose regimens that have demonstrated superiority compared to episodic treatment. […] Prophylaxis has significantly changed the lives of many hemophilia patients. […] Low and very-low dose prophylaxis and individualizing it to what is affordable for a given patient or nation, may bridge this gap created by global economic disparity.

• #16 NUWIQ® Personalized Prophylaxis | Optimizing Hemophilia A Treatmentnuwiq-logo

  https://nuwiqusa.com/hcp/dosing/personalized-prophylaxis/

  Optimizing FVIII therapy can be challenging—balancing dose and dosing frequency to prevent or reduce bleeding, while minimizing side effects, increasing convenience, and controlling overall treatment costs. Every hemophilia patient is unique, so a “one size fits all” approach to FVIII prophylaxis is not ideal. […] Personalized prophylaxis, whereby FVIII dose and dosing frequency is tailored to a patient’s individual pharmacokinetic data, has the potential to optimize care and enable fewer infusions. […] The NuPreviq clinical study showed that personalized prophylaxis with NUWIQ is effective. […] Personalized prophylaxis has the potential to optimize care and enable fewer infusions. […] Personalized Prophylaxis with NUWIQ Enabled the Majority of Patients to Extend Their Dosing Interval to Twice Weekly or Less.

• #17 Optimization of prophylaxis for hemophilia A | PLOS One

  https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0192783

  Prophylactic injections of factor VIII reduce the incidence of bleeds and slow the development of joint damage in people with hemophilia. The aim of this study was to identify optimal person-specific prophylaxis regimens for children with hemophilia A. […] The methods described here can be used to identify optimal, person-specific prophylaxis regimens for children with hemophilia A. […] Prophylactic intravenous injections of factor VIII reduce the incidence of bleeds and slow the development of joint damage in children with severe hemophilia A. […] While there has been extensive discussion of personalised prophylaxis there has not, to our knowledge, been a systematic effort to identify which of all possible prophylaxis regimens is optimal for an individual. […] Here we describe novel analytical and numerical methods to identify person-specific, globally optimal prophylaxis regimens for children with hemophilia A.

• #18 Pharmacokinetic model-based assessment of factor IX prophylaxis treatment regimens in severe hemophilia B | Scientific Reports

  https://www.nature.com/articles/s41598-024-70784-x

  An important aspect of improving care for people with hemophilia B (HB) is developing optimal treatment strategies. […] Prophylaxis with coagulation factor IX (FIX) replacement therapy is recommended to prevent bleeding and musculoskeletal damage due to FIX deficiency. […] Factor replacement therapy can be administered as standard half-life (SHL) or extended half-life (EHL) recombinant FIX (rFIX). EHL therapies are increasingly recommended in guidelines, as they require fewer infusions to achieve high factor levels with longer dosing intervals, which reduces the treatment burden compared with SHL products. […] Furthermore, EHLs are associated with improved bleeding prevention, quality of life and patient adherence, compared with SHL therapies. […] A comparison of potential target trough level attainability is clinically useful when evaluating available FIX therapies.

• #19 Hemophilia B (Factor IX Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779434-treatment

  The main goal of prophylactic treatment is to prevent bleeding symptoms and organ damage, in particular to joints. […] Hemophilic arthropathy that results from recurrent or target joint bleeding can be prevented by this method. […] In patients with congenital hemophilia B being treated prophylactically, a literature review by Franchini et al found the pooled, estimated, mean annualized bleeding rate (ABR) to be significantly lower in individuals who received recombinant FIX with extended half-life than in persons managed with recombinant FIX with standard half-life (1.29 vs 3.12, respectively). […] Prophylaxis is not universally accepted, with only about one third of children with hemophilia B receiving this treatment modality in the United States. […] Consensus recommendations on the management of hemophilia B by an international group of hematology experts include the following with regard to prophylaxis and factor treatment choice: Consider FIX prophylaxis in all people with severe hemophilia B, including those who have non-severe disease according to their basal FIX levels but with a severe bleeding phenotype; initiate prophylaxis as early as possible (ie, before the onset of joint bleeding) and do not interrupt it.

• #20 Pharmacokinetic model-based assessment of factor IX prophylaxis treatment regimens in severe hemophilia B | Scientific Reports

  https://www.nature.com/articles/s41598-024-70784-x

  An important aspect of improving care for people with hemophilia B (HB) is developing optimal treatment strategies. […] Prophylaxis with coagulation factor IX (FIX) replacement therapy is recommended to prevent bleeding and musculoskeletal damage due to FIX deficiency. […] Factor replacement therapy can be administered as standard half-life (SHL) or extended half-life (EHL) recombinant FIX (rFIX). EHL therapies are increasingly recommended in guidelines, as they require fewer infusions to achieve high factor levels with longer dosing intervals, which reduces the treatment burden compared with SHL products. […] Furthermore, EHLs are associated with improved bleeding prevention, quality of life and patient adherence, compared with SHL therapies. […] A comparison of potential target trough level attainability is clinically useful when evaluating available FIX therapies.

• #21 Hemophilia A (Factor VIII Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779322-treatment

  The main goal of prophylactic treatment is to prevent bleeding symptoms and organ damage, in particular to joints. Hemophilia arthropathy that results from recurrent or target joint bleeding can be prevented by this method. […] In 2014 the FDA approved a long-acting recombinant FVIIIFc fusion protein (rFVIIIFc) product (Eloctate) for control of bleeding episodes, management of perioperative bleeding, and routine prophylaxis in patients with hemophilia A. […] The rFVIIIFc product was developed by fusing rFVIII to the Fc portion of IgG1, which allows a naturally occurring pathway to prolong the product’s duration of action. FDA approval was based on a study in 164 patients with hemophilia A in which the median rate of bleeding episodes with prophylactic use of rFVIIIFc was 1.6 per year, compared with 33.6 per year in patients receiving on-demand treatment. […] The bispecific monoclonal antibody emicizumab is approved for prophylaxis in adults and children (including newborns) who have hemophilia A with or without FVIII inhibitors.

• #22

  https://haematologica.org/article/view/7323

  Early prophylaxis may require placing a central venous access device (CVAD) to facilitate frequent venous access, but these devices carry a risk of infections and thrombotic complications. In an attempt to reduce the need for CVADs while initiating early prophylaxis, Petrini and colleagues started prophylaxis with once-weekly infusions; many authors have subsequently published or recommended protocols starting with once-weekly infusions. […] The present study assesses how the increasing awareness of the importance of early prophylaxis affected bleeding before prophylaxis, CVAD use, initial prophylactic regimens, and achievement of primary prophylaxis.

• #23 Innovative Treatment Approaches in Routine Prophylaxis for Hemophilia A and B – Hematology Advisor

  https://www.hematologyadvisor.com/cch/hemophilia-a-b-prophylaxis-fviii-fix-marstacimab-emicizumab-concizumab/

  Methods that target tissue factor pathway inhibitor (TFPI) aim to enhance thrombin production and improve clotting by diminishing TFPI activity and thus facilitating more effective clot formation. […] Innovations are focusing on subcutaneous administration of nonfactor treatments, which offer longer half-lives and improve patient adherence through simplified treatment regimens. […] Clinical trials of marstacimab have shown a 92% reduction in bleeding rates and no thrombotic events. Potential Food and Drug Administration (FDA) approval is pending. […] As a leading nonfactor treatment for hemophilia A, emicizumab is widely prescribed and effective. […] No nonfactor treatment option is available for hemophilia B. Thus, patients with inhibitors are vulnerable to severe bleeding episodes.

• #24 Past, present & future of hemophilia treatment: from prophylaxis to gene therapies | VJHemOnc

  https://www.vjhemonc.com/feature/past-present-future-of-hemophilia-treatment-from-prophylaxis-to-gene-therapies/

  Past, present future of hemophilia treatment: from prophylaxis to gene therapies […] The treatment landscape for hemophilia is expanding. Improvements to established therapies and the development of novel strategies, such as the recently FDA approved gene therapies, are relieving the treatment burden of prophylaxis and improving patient lives. […] Treatment for hemophilia is typically prophylactic, preventing bleeds by allowing clot formation. This can be achieved through factor replacement therapies, non-replacement therapies or, more recently, gene therapies. […] Prophylactic factor replacement therapy is recognized as the standard of care for hemophilia, where the missing clotting factor is replaced by regular intravenous administration of a plasma-derived (pd) or recombinant factor (r).

• #25 Past, present & future of hemophilia treatment: from prophylaxis to gene therapies | VJHemOnc

  https://www.vjhemonc.com/feature/past-present-future-of-hemophilia-treatment-from-prophylaxis-to-gene-therapies/

  Emicizumab was approved by the FDA for patients with hemophilia A with and without inhibitors in 2018. This was based on the Phase III HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160) trials, where treatment reduced the ABR of patients with and without inhibitors compared to no prophylaxis. […] The novel approaches discussed here increase treatment options for patients with hemophilia who develop inhibitors from factor replacement therapy, or who cannot afford or manage weekly infusions. Novel non-replacement therapies are effective for patients with hemophilia A and B regardless of their inhibitor status, and the recently-approved AAV-based gene therapies offer the potential for a one-time treatment for hemophilia A and B. […] While there are still hurdles regarding the treatment and financial burden on patients, the therapeutic landscape for hemophilia is constantly evolving and improving.

• #26 Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A | Radhakrishnan | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/1346/870

  Patients with hemophilia need lifelong access to clotting factor concentrates, either as replacement for preventing or as episodic for treating bleeding. […] Emicizumab, a bispecific monoclonal antibody which binds to factor IXa and X and activates the latter has been approved for use in patients with inhibitor-positive hemophilia A (HAI). […] The study demonstrates how monoclonal agents that are currently licensed as well as those in the pipeline will be the game changer in hemophilia care, offering not only the advantage of subcutaneous products and less hospital visits, but also near-zero bleeds even at lower-dose range. It offsets not only the direct costs as has been demonstrated here, but also reduces the out-of-pocket expenses of the family by reducing hospital visits and medical infrastructure costs due to subcutaneous administration, lesser or probably no need for joint replacement surgeries in the future for these children. It also offers the promise of better school and job attendance and lesser chance of disability, as well as more employment opportunities for these patients.

• #27 Innovative Treatment Approaches in Routine Prophylaxis for Hemophilia A and B – Hematology Advisor

  https://www.hematologyadvisor.com/cch/hemophilia-a-b-prophylaxis-fviii-fix-marstacimab-emicizumab-concizumab/

  Recent advances in hemophilia treatment have revealed promising strategies for improving patient outcomes while minimizing the therapeutic burden. […] Marstacimab has shown considerable promise in clinical trials, achieving statistically significant and clinically meaningful reductions in annualized bleeding rates compared with conventional prophylaxis and on-demand intravenous treatments. […] Another promising therapy is concizumab, a monoclonal antibody that also targets TFPI to enhance thrombin generation. […] Innovative nonfactor treatments are undergoing exploration for subcutaneous administration, and they demonstrate significantly longer half-lives than current factor therapies. […] Unmet needs include the development of subcutaneous and self-administered options to improve patient access and adherence, along with the management of side effects such as injection site reactions.

• #28 Innovative Treatment Approaches in Routine Prophylaxis for Hemophilia A and B – Hematology Advisor

  https://www.hematologyadvisor.com/cch/hemophilia-a-b-prophylaxis-fviii-fix-marstacimab-emicizumab-concizumab/

  TFPI-targeting methods represent promising strategies to enhance thrombin production and improve clotting in patients with bleeding disorders, particularly hemophilia. […] The value of TFPI inhibitors lies in their ability to provide effective long-term prophylaxis and address the challenges faced by patients with inhibitors, making them an important addition to the hemophilia treatment landscape. […] These rebalancing therapies are under investigation in cohorts of patients with hemophilia A or B, a novel approach relative to previous treatments that were specifically developed for a single type of hemophilia.

• #29 FDA Approves New Treatment for Hemophilia A or B | FDA

  https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-hemophilia-or-b

  Today, the U.S. Food and Drug Administration approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies). […] Hympavzi is a new type of drug that, rather than replacing a clotting factor, works by reducing the amount, and therefore, the activity of, the naturally occurring anticoagulation protein called tissue factor pathway inhibitor. This increases the amount of thrombin, an enzyme that is critical in blood clotting, that is generated. This is expected to reduce or prevent the frequency of bleeding episodes. […] In the initial six-month period during which patients received prophylactic factor replacement, the estimated annualized bleeding rate was 7.85 and was 5.08 during the subsequent 12 months on Hympavzi prophylaxis, showing that Hympavzi provided similar bleeding rates.

• #30 Innovative Treatment Approaches in Routine Prophylaxis for Hemophilia A and B – Hematology Advisor

  https://www.hematologyadvisor.com/cch/hemophilia-a-b-prophylaxis-fviii-fix-marstacimab-emicizumab-concizumab/

  Recent advances in hemophilia treatment have revealed promising strategies for improving patient outcomes while minimizing the therapeutic burden. […] Marstacimab has shown considerable promise in clinical trials, achieving statistically significant and clinically meaningful reductions in annualized bleeding rates compared with conventional prophylaxis and on-demand intravenous treatments. […] Another promising therapy is concizumab, a monoclonal antibody that also targets TFPI to enhance thrombin generation. […] Innovative nonfactor treatments are undergoing exploration for subcutaneous administration, and they demonstrate significantly longer half-lives than current factor therapies. […] Unmet needs include the development of subcutaneous and self-administered options to improve patient access and adherence, along with the management of side effects such as injection site reactions.

• #31 Past, present & future of hemophilia treatment: from prophylaxis to gene therapies | VJHemOnc

  https://www.vjhemonc.com/feature/past-present-future-of-hemophilia-treatment-from-prophylaxis-to-gene-therapies/

  Emicizumab was approved by the FDA for patients with hemophilia A with and without inhibitors in 2018. This was based on the Phase III HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160) trials, where treatment reduced the ABR of patients with and without inhibitors compared to no prophylaxis. […] The novel approaches discussed here increase treatment options for patients with hemophilia who develop inhibitors from factor replacement therapy, or who cannot afford or manage weekly infusions. Novel non-replacement therapies are effective for patients with hemophilia A and B regardless of their inhibitor status, and the recently-approved AAV-based gene therapies offer the potential for a one-time treatment for hemophilia A and B. […] While there are still hurdles regarding the treatment and financial burden on patients, the therapeutic landscape for hemophilia is constantly evolving and improving.

• #32 Hemophilia B (Factor IX Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779434-treatment

  A cost-benefit analysis indicates that this approach reduces overall factor use and significantly reduces morbidity. […] Dosing is designed to maintain trough levels greater than 2%. […] With the development of FIX preparations that have extended half-lives, dosing for routine prophylaxis may be as infrequent as every 10 days. […] An option for prophylaxis is marstacimab, a monoclonal antibody directed against tissue factor pathway inhibitor, which was approved by the US Food and Drug Administration (FDA) in October 2024. […] Another such option is fitusiran, an antithrombin-directed small interfering ribonucleic acid (RNA), which received FDA approval in March 2025. […] Gene therapy offers the potential for a definitive cure and has entered clinical practice, with FDA approval of the first product in 2022.

• #33 Past, present & future of hemophilia treatment: from prophylaxis to gene therapies | VJHemOnc

  https://www.vjhemonc.com/feature/past-present-future-of-hemophilia-treatment-from-prophylaxis-to-gene-therapies/

  Past, present future of hemophilia treatment: from prophylaxis to gene therapies […] The treatment landscape for hemophilia is expanding. Improvements to established therapies and the development of novel strategies, such as the recently FDA approved gene therapies, are relieving the treatment burden of prophylaxis and improving patient lives. […] Treatment for hemophilia is typically prophylactic, preventing bleeds by allowing clot formation. This can be achieved through factor replacement therapies, non-replacement therapies or, more recently, gene therapies. […] Prophylactic factor replacement therapy is recognized as the standard of care for hemophilia, where the missing clotting factor is replaced by regular intravenous administration of a plasma-derived (pd) or recombinant factor (r).

• #34 UCSF Hemophilia Trial → Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

  https://clinicaltrials.ucsf.edu/trial/NCT06379789

  Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of a gene into your DNA) called REGV131-LNP1265, also called the „study drug”. Gene insertion therapy aims to teach the body how to produce clotting factor long-term, without the need for factor replacement therapy. […] The main aim of this study is to find a safe and well-tolerated dose of the study drug by checking the side effects that may happen from taking it. […] How often factor replacement therapy is needed, both on a regular basis for prevention of bleeding, and as needed to treat bleeding events (and it if changes after taking study drug)

• #35 Hemophilia B (Factor IX Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779434-treatment

  A cost-benefit analysis indicates that this approach reduces overall factor use and significantly reduces morbidity. […] Dosing is designed to maintain trough levels greater than 2%. […] With the development of FIX preparations that have extended half-lives, dosing for routine prophylaxis may be as infrequent as every 10 days. […] An option for prophylaxis is marstacimab, a monoclonal antibody directed against tissue factor pathway inhibitor, which was approved by the US Food and Drug Administration (FDA) in October 2024. […] Another such option is fitusiran, an antithrombin-directed small interfering ribonucleic acid (RNA), which received FDA approval in March 2025. […] Gene therapy offers the potential for a definitive cure and has entered clinical practice, with FDA approval of the first product in 2022.

• #36 Past, present & future of hemophilia treatment: from prophylaxis to gene therapies | VJHemOnc

  https://www.vjhemonc.com/feature/past-present-future-of-hemophilia-treatment-from-prophylaxis-to-gene-therapies/

  Emicizumab was approved by the FDA for patients with hemophilia A with and without inhibitors in 2018. This was based on the Phase III HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160) trials, where treatment reduced the ABR of patients with and without inhibitors compared to no prophylaxis. […] The novel approaches discussed here increase treatment options for patients with hemophilia who develop inhibitors from factor replacement therapy, or who cannot afford or manage weekly infusions. Novel non-replacement therapies are effective for patients with hemophilia A and B regardless of their inhibitor status, and the recently-approved AAV-based gene therapies offer the potential for a one-time treatment for hemophilia A and B. […] While there are still hurdles regarding the treatment and financial burden on patients, the therapeutic landscape for hemophilia is constantly evolving and improving.

• #37

  https://www.roche.com/stories/prophylaxis-in-haemophilia

  Prophylaxis now considered the standard of care in haemophilia A treatment can on average, cut down bleeds from more than 40 times a year to less than two bleeds a year. Prophylaxis can protect against spontaneous bleeds, such as joint bleeds, as well as potentially life-threatening bleeds such as those in the head or gastrointestinal tract. Adherence to a prophylaxis protocol is critical to its success to maintain appropriate clotting levels in the blood. […] The change from on-demand treatment when a bleed occurs to prophylaxis to prevent bleeds was an important shift in haemophilia care. […] The case for starting prophylaxis as early as possible to avoid any joint damage is clear, she says. […] Since the introduction of prophylaxis in the 1990s, people with haemophilia A have seen a dramatic reduction in the frequency of bleeds, which has had a huge impact on their day-to-day lives.

• #38 Hemophilia A (Factor VIII Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779322-treatment

  The main goal of prophylactic treatment is to prevent bleeding symptoms and organ damage, in particular to joints. Hemophilia arthropathy that results from recurrent or target joint bleeding can be prevented by this method. […] In 2014 the FDA approved a long-acting recombinant FVIIIFc fusion protein (rFVIIIFc) product (Eloctate) for control of bleeding episodes, management of perioperative bleeding, and routine prophylaxis in patients with hemophilia A. […] The rFVIIIFc product was developed by fusing rFVIII to the Fc portion of IgG1, which allows a naturally occurring pathway to prolong the product’s duration of action. FDA approval was based on a study in 164 patients with hemophilia A in which the median rate of bleeding episodes with prophylactic use of rFVIIIFc was 1.6 per year, compared with 33.6 per year in patients receiving on-demand treatment. […] The bispecific monoclonal antibody emicizumab is approved for prophylaxis in adults and children (including newborns) who have hemophilia A with or without FVIII inhibitors.

• #39 Hemophilia A (Factor VIII Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779322-treatment

  The main goal of prophylactic treatment is to prevent bleeding symptoms and organ damage, in particular to joints. Hemophilia arthropathy that results from recurrent or target joint bleeding can be prevented by this method. […] In 2014 the FDA approved a long-acting recombinant FVIIIFc fusion protein (rFVIIIFc) product (Eloctate) for control of bleeding episodes, management of perioperative bleeding, and routine prophylaxis in patients with hemophilia A. […] The rFVIIIFc product was developed by fusing rFVIII to the Fc portion of IgG1, which allows a naturally occurring pathway to prolong the product’s duration of action. FDA approval was based on a study in 164 patients with hemophilia A in which the median rate of bleeding episodes with prophylactic use of rFVIIIFc was 1.6 per year, compared with 33.6 per year in patients receiving on-demand treatment. […] The bispecific monoclonal antibody emicizumab is approved for prophylaxis in adults and children (including newborns) who have hemophilia A with or without FVIII inhibitors.

• #40 Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A | Radhakrishnan | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/1346/870

  Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A […] Background: The real-world data on outcome of hemophilia A patients with inhibitors (HAI) is sparse, especially from developing countries. In a setting of inequitable healthcare opportunities for hemophilia patients, especially those with inhibitors, low-dose practices of emicizumab are emerging. In the present article, we describe our experience of managing HAI patients on low-dose emicizumab over a period of 56 months (from December 2019 to August 2024). […] Methods: The present study reports the response of patients with inhibitor-positive severe hemophilia A (HAI) and a high annual bleed rate to two-dose schedules of emicizumab prophylaxis. All patients with HAI were previously managed with on-demand bypassing agents (BPAs) before being shifted to emicizumab. Seven patients were treated on standard dose of 3 mg/kg weekly for 4 weeks followed by once in 2 weeks, whereas 25 patients were started on low dose of 3 mg/kg once in 4 weeks with or without loading as per clinical decision. […] Emicizumab prophylaxis even in lower doses is effective in preventing bleeds and improving joint outcome in HAI with pre-existing high bleed rate and arthropathy. This opens up an avenue for providing equity in healthcare delivery for HAI in low- and middle-income countries (LMICs) such as India.

• #41 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  The first RCT on prophylaxis vs. on-demand treatment, the Joint Outcome Study (JOS), was published in August 2007. […] This study, whose primary end-point focused on the prevention of joint deterioration by prophylaxis started prior to or at the time of the second joint bleed (between 6 and 30 months old) provided evidence of an about 6-fold reduction (85%) in the risk of joint damage in children on prophylaxis compared to those on intensive on-demand treatment. […] Interestingly, only MRI assessment showed a significant difference in the joint outcome, with more than half of the joint abnormalities not being detectable by the traditional radiological evaluation. […] Recent data suggest new potential benefits of early initiation of prophylaxis in haemophilic children. A protective effect against the development of inhibitors has been shown in a case-control Italian study, with a 70% reduction of inhibitor risk in children starting prophylaxis at a median age of 35 months.

• #42 Hemophilia Prophylaxis Leads to Joint Health | HemAware

  https://hemaware.org/bleeding-disorders-z/hemophilia-prophylaxis-leads-joint-health

  Preventing joint damage from repeated bleeds […] Hemophilia prophylaxis since he was four years old. […] For more than 40 years, doctors who care for people with hemophilia have known that therapy with replacement clotting factor can reduce bleeding into joints, lowering the risk of a common and troublesome complication of bleeding disorders—the joint deterioration that results from repeated bleeds. […] For the first time, a well-controlled, randomized clinical trial showed that regular prophylaxis with recombinant factor VIII can prevent joint disease in young boys with severe hemophilia A. […] This strengthens the argument for prophylaxis. […] Although the benefits of hemophilia prophylaxis seemed increasingly apparent, other forces have worked against its wider practice in the US.

• #43 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  The first RCT on prophylaxis vs. on-demand treatment, the Joint Outcome Study (JOS), was published in August 2007. […] This study, whose primary end-point focused on the prevention of joint deterioration by prophylaxis started prior to or at the time of the second joint bleed (between 6 and 30 months old) provided evidence of an about 6-fold reduction (85%) in the risk of joint damage in children on prophylaxis compared to those on intensive on-demand treatment. […] Interestingly, only MRI assessment showed a significant difference in the joint outcome, with more than half of the joint abnormalities not being detectable by the traditional radiological evaluation. […] Recent data suggest new potential benefits of early initiation of prophylaxis in haemophilic children. A protective effect against the development of inhibitors has been shown in a case-control Italian study, with a 70% reduction of inhibitor risk in children starting prophylaxis at a median age of 35 months.

• #44 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  On the whole, severe haemophiliacs on prophylaxis and their families have a better quality of life than patients who receive on-demand treatment. […] The definitions of prophylaxis, therefore, reflect a wide spectrum of clinical conditions and objectives of the treatment, from the prevention of severe or life-threatening bleeds to the absence of arthropathy enabling patients to live a substantially normal life, without overprotection. […] In a congenital, chronic disease such as haemophilia, for which a definitive cure is still not available, the latter perspective is an important and reliable goal, achieved by primary prophylaxis. […] The key point is, therefore, likely to be the number of joint bleeds before the start of prophylaxis, as also shown in the Dutch experience with lower dose regimens, tailored to the patients bleeding tendency.

• #45

• #46 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  In patients with severe haemophilia, prophylaxis, i.e. the infusion of factor concentrate replacement treatment in order to prevent bleeding episodes, has been used for more than 40 years in Northern Europe and more recently in other European countries and in Northern America, becoming the first-choice treatment recommended by the World Health Organisation (WHO) and the World Federation of Haemophilia since 1994. […] The main objective of the prophylactic replacement treatment since the Swedish pioneering regimens was (and remains) to minimise the number of joint bleeds since an early age by converting the severe form of haemophilia to a milder form. […] Preventing or reducing the clinical impact of haemophilic arthropathy by prophylaxis means enabling normal life and psychosocial development for haemophilic children, including the possibility of physical activities, regular school attendance and, consequently, social and work opportunities.

• #47 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  On the whole, severe haemophiliacs on prophylaxis and their families have a better quality of life than patients who receive on-demand treatment. […] The definitions of prophylaxis, therefore, reflect a wide spectrum of clinical conditions and objectives of the treatment, from the prevention of severe or life-threatening bleeds to the absence of arthropathy enabling patients to live a substantially normal life, without overprotection. […] In a congenital, chronic disease such as haemophilia, for which a definitive cure is still not available, the latter perspective is an important and reliable goal, achieved by primary prophylaxis. […] The key point is, therefore, likely to be the number of joint bleeds before the start of prophylaxis, as also shown in the Dutch experience with lower dose regimens, tailored to the patients bleeding tendency.

• #48 Low dose long-acting factor VIII prophylaxis in pediatric and young adult patients with hemophilia A: Short-term single-center experience from a developing country – Journal of Hematology and Allied Sciences

  https://jhas-bsh.com/low-dose-long-acting-factor-viii-prophylaxis-in-pediatric-and-young-adult-patients-with-hemophilia-a-short-term-single-center-experience-from-a-developing-country/

  In this study, we demonstrated that low to medium dose (20 IU/Kg BW twice a week) long-acting factor VIII-Fc concentrate given as secondary/tertiary prophylaxis can result in 88.9%, 89%, and 88.9% reduction in total number of bleeds, total number of joint bleeds, and ABR, respectively, in pediatric and young adult patients with hemophilia A. […] Long-acting factor VIII formulation (rFVIII-Fc, Eloctate) has a promising role in prophylaxis for hemophilia patients. Low dose, twice a week prophylaxis with this agent can be a reasonable option for patients with hemophilia A in developing countries. It significantly reduces joint bleeds, overall bleeds, school absenteeism, emergency visits, and improves joint scores without risk of inhibitor formation and any significant adverse events.

• #49 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  On the whole, severe haemophiliacs on prophylaxis and their families have a better quality of life than patients who receive on-demand treatment. […] The definitions of prophylaxis, therefore, reflect a wide spectrum of clinical conditions and objectives of the treatment, from the prevention of severe or life-threatening bleeds to the absence of arthropathy enabling patients to live a substantially normal life, without overprotection. […] In a congenital, chronic disease such as haemophilia, for which a definitive cure is still not available, the latter perspective is an important and reliable goal, achieved by primary prophylaxis. […] The key point is, therefore, likely to be the number of joint bleeds before the start of prophylaxis, as also shown in the Dutch experience with lower dose regimens, tailored to the patients bleeding tendency.

• #50 Hemophilia A (Factor VIII Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779322-treatment

  In most developed countries with access to recombinant product, prophylaxis is primary (ie, therapy is started in patients as young as 1 y and continues into adolescence). A cost-benefit analysis indicates that this approach reduces overall factor use and significantly reduces morbidity. […] For prophylaxis, dosing is designed to maintain trough levels of 2% or higher. This usually requires the administration of FVIII three times per week. Individualized therapy (ie, tailored prophylaxis) has been also used with success; the best approach has yet to be determined. Monoclonal antibodies (ie, emicizumab, marstacimab) may also be used for prophylaxis. […] Most of the care for children with severe hemophilia now takes place at home, in the community, and at school, allowing these children to participate in normal activities that are otherwise impossible. This approach resulted from the development of prophylactic regimens of factor concentrate infusions that are administered at home, usually by a parent.

• #51

  https://link.springer.com/article/10.1007/s40801-024-00452-z

  While initial costs increase, prophylaxis becomes increasingly cost effective over time due to the prevention of bleeding episodes. […] Prophylactic treatment has been found to be more cost effective than on-demand treatment in numerous studies. […] The concept of prophylactic hemophilia treatment, which involves the regular infusion of factor concentrates, was first introduced in the 1950s. […] Studies have shown that prophylactic treatment is efficacious in reducing the incidence of severe bleeding events and the severity of hemophilic arthropathy in patients with severe HA. […] Primary prophylaxis, defined as regular factor treatment starting before any joint damage caused by repeated bleeding has occurred, has become the standard of care for patients with severe hemophilia because it is associated with a better health-related quality of life (HRQoL) than on-demand treatment.

• #52 How to give Haemophilia patients a fuller life – India Today

  https://www.indiatoday.in/india-today-insight/story/how-to-give-haemophilia-patients-a-fuller-life-2718781-2025-05-02

  Haemophilia, a rare genetic bleeding disorder in which the blood doesnt clot properly, affects over 100,000 people in India. […] Most Haemophilia patients in India rely on on-demand therapygetting treatment only after bleeding starts. This reactive approach means more than 20 painful bleeds a year; about 12 of these bleeds happen in the joints, such as knees and elbows, each episode requiring hospitalisation and recovery time. […] Prophylaxis, the alternative treatment, can transform the situation. This preventive treatment involves regular infusions to stop bleeds before they occur. Globally, its the gold standard that reduces bleeds by more than half and allows people to live near-normal lives. […] While prophylaxis costs more upfront, its actually cheaper in the long term when you factor in emergency care costs and lost productivity. […] Every year of prophylaxis saves over 15 productive days per bleed. Early treatment prevents disabilities, which can cost the nation far more in welfare and lost productivity.

• #53

  https://link.springer.com/article/10.1007/s40801-024-00452-z

  Prophylactic treatment improved quality of life and was a cost-effective option. […] Our study demonstrated that intermediate-dose FVIII prophylaxis is a highly effective treatment for severe hemophilia A in Taiwan. […] To achieve zero bleeding events and improve clinical outcomes even further, developing an individualized treatment plan based on the patients age, bleeding phenotype, physical activity, specific clinical scenario, and pharmacokinetic profile is essential. […] Prophylactic treatment should be tailored to each patient, and many patients may do well on lower doses of FVIII. […] This study compared the cost effectiveness of intermediate-dose prophylaxis treatment and reactive treatment with comparable outcomes shown with other studies, which can assist health policymakers in determining which regimen to pay for when working with a limited budget.

• #54 Individualized prophylaxis for optimizing hemophilia care: can we apply this to both developed and developing nations? | Thrombosis Journal | Full Text

  https://thrombosisjournal.biomedcentral.com/articles/10.1186/s12959-016-0096-y

  Prophylaxis is considered optimal care for hemophilia patients to prevent bleeding and to preserve joint function thereby improving quality of life (QoL). […] Prophylaxis can be further individualized to improve outcomes and cost effectiveness. […] Prophylaxis in economically constrained nations is limited by the ill-affordability of clotting factor concentrates. […] However, at least 5 studies on children and adults from Thailand, China and India have shown superiority of low dose (~510 IU kg1 2-3 per week) prophylaxis over episodic treatment in terms of bleed reduction, and quality of life, with improved physical activity, independent functioning, school attendance and community participation. […] Prophylaxis must be individualized to include affordability as a major determinant in order to optimize low-dose regimens that have demonstrated superiority compared to episodic treatment. […] Prophylaxis has significantly changed the lives of many hemophilia patients. […] Low and very-low dose prophylaxis and individualizing it to what is affordable for a given patient or nation, may bridge this gap created by global economic disparity.

• #55

  https://link.springer.com/article/10.1007/s12288-019-01147-0

  Hemophilia is an inherited bleeding disorder which causes impaired blood clotting. The currently accepted standard of care of this disease is prophylaxis therapy (PT) with CFC. Prophylaxis therapy for Hemophilia is given in developed countries for the last few decades. […] Several studies conducted in developed countries have confirmed the clinical efficacy of PT in Hemophilia treatment. Currently, there exist several challenges for implementation of this effective treatment in resource poor nations. Low dose prophylaxis (LDP) has been developed as a solution to minimize these challenges and to provide better care for subjects with Hemophilia from low resource countries. The impact of LDP was evaluated by several recent studies and the reported clinical outcomes seem to suggest an optimistic future for this line of therapy.

• #56 NUWIQ® Personalized Prophylaxis | Optimizing Hemophilia A Treatmentnuwiq-logo

  https://nuwiqusa.com/hcp/dosing/personalized-prophylaxis/

  Optimizing FVIII therapy can be challenging—balancing dose and dosing frequency to prevent or reduce bleeding, while minimizing side effects, increasing convenience, and controlling overall treatment costs. Every hemophilia patient is unique, so a “one size fits all” approach to FVIII prophylaxis is not ideal. […] Personalized prophylaxis, whereby FVIII dose and dosing frequency is tailored to a patient’s individual pharmacokinetic data, has the potential to optimize care and enable fewer infusions. […] The NuPreviq clinical study showed that personalized prophylaxis with NUWIQ is effective. […] Personalized prophylaxis has the potential to optimize care and enable fewer infusions. […] Personalized Prophylaxis with NUWIQ Enabled the Majority of Patients to Extend Their Dosing Interval to Twice Weekly or Less.

• #57 Optimization of prophylaxis for hemophilia A | PLOS One

  https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0192783

  Prophylactic injections of factor VIII reduce the incidence of bleeds and slow the development of joint damage in people with hemophilia. The aim of this study was to identify optimal person-specific prophylaxis regimens for children with hemophilia A. […] The methods described here can be used to identify optimal, person-specific prophylaxis regimens for children with hemophilia A. […] Prophylactic intravenous injections of factor VIII reduce the incidence of bleeds and slow the development of joint damage in children with severe hemophilia A. […] While there has been extensive discussion of personalised prophylaxis there has not, to our knowledge, been a systematic effort to identify which of all possible prophylaxis regimens is optimal for an individual. […] Here we describe novel analytical and numerical methods to identify person-specific, globally optimal prophylaxis regimens for children with hemophilia A.

• #58 Hemophilia B (Factor IX Deficiency) Treatment & Management: Approach Considerations, Prehospital Care, Emergency Department Care

  https://emedicine.medscape.com/article/779434-treatment

  The main goal of prophylactic treatment is to prevent bleeding symptoms and organ damage, in particular to joints. […] Hemophilic arthropathy that results from recurrent or target joint bleeding can be prevented by this method. […] In patients with congenital hemophilia B being treated prophylactically, a literature review by Franchini et al found the pooled, estimated, mean annualized bleeding rate (ABR) to be significantly lower in individuals who received recombinant FIX with extended half-life than in persons managed with recombinant FIX with standard half-life (1.29 vs 3.12, respectively). […] Prophylaxis is not universally accepted, with only about one third of children with hemophilia B receiving this treatment modality in the United States. […] Consensus recommendations on the management of hemophilia B by an international group of hematology experts include the following with regard to prophylaxis and factor treatment choice: Consider FIX prophylaxis in all people with severe hemophilia B, including those who have non-severe disease according to their basal FIX levels but with a severe bleeding phenotype; initiate prophylaxis as early as possible (ie, before the onset of joint bleeding) and do not interrupt it.

• #59 Considerations for Prophylaxis in Patients With Hemophilia A

  https://www.pharmacytimes.com/view/considerations-for-prophylaxis-in-patients-with-hemophilia-a

  Key opinion leaders discuss the factors in giving patients with hemophilia A prophylaxis, including any barriers and discussions with the patient. […] Well often talk about continuous prophylaxis, this idea of giving it on a regular basis. […] The guidelines have come out and said that even in moderate patients, if they have a severe bleeding phenotype, they should be offered prophylaxis as well. […] At least half of our moderate patients, those with levels of 1% to 5%, are on prophylaxis for at least some period of time before the age of 18. […] However, typically in a young child, you cant start out maybe at 3 times a week, every other day, or every 48 hours or every 72 hours. […] With prophylaxis, as were looking into the future and playing the long game, in an ideal state we would be continuing and reaping the benefits of the prophylaxis long term.

• #60

  https://haematologica.org/article/view/9730

  During factor VIII prophylaxis for severe hemophilia A, bleeding risk increases with time when factor VIII activity is below 1%. Maintaining trough activity above 1% does not protect all patients from bleeding. […] The risk of spontaneous bleeds during periods with FVIII:C 1% in subjects on prophylaxis is unknown, although an epidemiological study in individuals with mild/moderate hemophilia suggested that maintaining FVIII:C above 1% will not protect all patients from bleeding. […] The use of a large number of data on outcomes and their potential modifiers, combined from patients in different age groups and trial phases, is a strength of our study. […] The choice of prophylactic regimens to target certain trough levels should take into account the patients age, joint disease activity, and other determinants of bleeding risk.

• #61 Considerations for Prophylaxis in Patients With Hemophilia A

  https://www.pharmacytimes.com/view/considerations-for-prophylaxis-in-patients-with-hemophilia-a

  Yes, in very individualized cases, absolutely, we would consider discontinuing prophylaxis if the burdens outweigh the benefits that are being realized in the individual. […] Its never going to be normal, but were trying to get them so they can participate in life and physical activities some sports, obviously noncontact. […] Patient preference is certainly important in the selection of treatment and should be considered in decision-making, so we can help make the optimal treatment choice for not only favorable outcomes but sustainability in terms of adherence. […] In terms of some patient preferences that we see at the pharmacy level, aside from cost, 1 of the concerns is for developing inhibitors against Factor VIII replacement products.

• #62 Hemophilia A and B Prophylaxis Recommendations | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-267-masac-recommendation-concerning-prophylaxis-for-hemophilia-a-and-b-with-and-without-inhibitors

  Prevention of early life bleeding including mitigation of the risk of intracranial hemorrhage may be possible if emicizumab prophylaxis is implemented prior to 6 months of age. […] Adherence with prophylaxis should be monitored. Options for monitoring include self-report, electronic diaries, and pharmacy reports. […] Regular surveillance and utilization of a multidisciplinary team approach are critical to ensuring adherence to a mutually decided prophylactic regimen. […] Shared decision-making tools are available to help guide health care professionals and patients to guide decision-making about prophylaxis.

• #63 Hemophilia A & B Prophylaxis – Guidelines Side-By-Side – Guideline Central

  https://www.guidelinecentral.com/insights/hemophilia-side-by-side/

  For patients who are adherent to their prescribed prophylaxis regimen but still experience breakthrough bleeds: Recommends escalation of prophylaxis with measurement of trough levels and, if required, orthopedic interventions as appropriate. […] For patients with severe phenotype hemophilia A or B on prophylaxis: Recommend patients caregivers be taught to maintain timely and accurate records of bleeding episodes and treatment and be followed in hemophilia treatment centers. […] For patients with severe and moderately-severe hemophilia A without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events. […] For patients with severe hemophilia A with inhibitors: Suggest prophylaxis over episodic treatment of bleeding events. […] For patients with severe and moderately-severe hemophilia B without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events.

• #64 Hemophilia A & B Prophylaxis – Guidelines Side-By-Side – Guideline Central

  https://www.guidelinecentral.com/insights/hemophilia-side-by-side/

  For patients who are adherent to their prescribed prophylaxis regimen but still experience breakthrough bleeds: Recommends escalation of prophylaxis with measurement of trough levels and, if required, orthopedic interventions as appropriate. […] For patients with severe phenotype hemophilia A or B on prophylaxis: Recommend patients caregivers be taught to maintain timely and accurate records of bleeding episodes and treatment and be followed in hemophilia treatment centers. […] For patients with severe and moderately-severe hemophilia A without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events. […] For patients with severe hemophilia A with inhibitors: Suggest prophylaxis over episodic treatment of bleeding events. […] For patients with severe and moderately-severe hemophilia B without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events.

• #65 Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review | Orphanet Journal of Rare Diseases | Full Text

  https://ojrd.biomedcentral.com/articles/10.1186/s13023-021-01919-w

  The present systematic literature review therefore summarizes factors (barriers and promoters) for adherence to prophylactic treatment in young adults, the effect of non-adherence to prophylaxis, the importance of prophylaxis in young adults, and the effect of different doses (low, medium, and standard dose) and durations of treatment (short and long term) on patients health. […] The adherence to prophylaxis was reported to be higher in patients below 16 years of age, which means that patients tend to discontinue prophylaxis while transitioning from childhood to adolescence. […] An Italian study reported high adherence to prophylaxis (80%) and increased physical activity in adherent patients. […] The majority of studies reported annualized bleeding rates (ABRs) varying from zero joint bleeds to 11.8 bleeds. All the studies have reported better ABRs, joint bleed rates, HRQoL with prophylaxis compared with on-demand treatment.

• #66 Hemophilia A & B Prophylaxis – Guidelines Side-By-Side – Guideline Central

  https://www.guidelinecentral.com/insights/hemophilia-side-by-side/

  For patients who are adherent to their prescribed prophylaxis regimen but still experience breakthrough bleeds: Recommends escalation of prophylaxis with measurement of trough levels and, if required, orthopedic interventions as appropriate. […] For patients with severe phenotype hemophilia A or B on prophylaxis: Recommend patients caregivers be taught to maintain timely and accurate records of bleeding episodes and treatment and be followed in hemophilia treatment centers. […] For patients with severe and moderately-severe hemophilia A without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events. […] For patients with severe hemophilia A with inhibitors: Suggest prophylaxis over episodic treatment of bleeding events. […] For patients with severe and moderately-severe hemophilia B without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events.

• #67 Hemophilia A and B Prophylaxis Recommendations | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-267-masac-recommendation-concerning-prophylaxis-for-hemophilia-a-and-b-with-and-without-inhibitors

  In view of the demonstrated benefits of prophylaxis begun at a young age in persons with hemophilia A or B (PwH), MASAC recommends that prophylaxis be considered standard of care therapy for individuals with severe hemophilia A or B (factor VIII or factor IX 1%) including those with inhibitors. Prophylactic therapy may also be considered for PwH with moderate and mild hemophilia with a severe phenotype. Prophylactic therapy should be instituted early (prior to the onset of frequent bleeding). […] Prophylaxis should be initiated at an early age, ideally before age 3 years and prior to the second joint bleed; prophylaxis may be considered within the first six months of life to reduce occurrence of intracranial hemorrhage. […] The goals for emicizumab prophylaxis remain like that of factor prophylaxis in that a regular administration of a prophylactic hemostatic agent should be considered as early as feasible to prevent the long-term complications of musculoskeletal bleeding and allow the patient to lead a physically active life approaching the quality of life of an unaffected individual.

• #68 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  Long-term clinical outcome was more favourable in patients starting prophylaxis before the age of 4 (no arthropathy in 50% of patients vs. 21% in those starting between 3 and 7 years old), with an estimated 8% higher Pettersson score per year of postponed prophylaxis from birth, and the best results (70% of patients with score 0) found in patients with fewer than three bleeds before the start of prophylaxis. […] The relative rarity of the disease and the difficulty in performing randomised controlled studies (especially in countries in which consolidated experience on prophylaxis raises ethical considerations on possible randomisation of children to on-demand treatment) until recently, led the Cochrane reviewers in 2005 to identify only four small randomised cross-over trials, comparing a prophylactic regimen with placebo or two different prophylactic regimens.

• #69

  https://haematologica.org/article/view/7323

  Early prophylaxis may require placing a central venous access device (CVAD) to facilitate frequent venous access, but these devices carry a risk of infections and thrombotic complications. In an attempt to reduce the need for CVADs while initiating early prophylaxis, Petrini and colleagues started prophylaxis with once-weekly infusions; many authors have subsequently published or recommended protocols starting with once-weekly infusions. […] The present study assesses how the increasing awareness of the importance of early prophylaxis affected bleeding before prophylaxis, CVAD use, initial prophylactic regimens, and achievement of primary prophylaxis.

• #70 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  The first RCT on prophylaxis vs. on-demand treatment, the Joint Outcome Study (JOS), was published in August 2007. […] This study, whose primary end-point focused on the prevention of joint deterioration by prophylaxis started prior to or at the time of the second joint bleed (between 6 and 30 months old) provided evidence of an about 6-fold reduction (85%) in the risk of joint damage in children on prophylaxis compared to those on intensive on-demand treatment. […] Interestingly, only MRI assessment showed a significant difference in the joint outcome, with more than half of the joint abnormalities not being detectable by the traditional radiological evaluation. […] Recent data suggest new potential benefits of early initiation of prophylaxis in haemophilic children. A protective effect against the development of inhibitors has been shown in a case-control Italian study, with a 70% reduction of inhibitor risk in children starting prophylaxis at a median age of 35 months.

• #71 Prophylaxis in haemophilia should be life-long

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3320774/

  Prophylaxis in haemophilia should be life-long. […] Although prophylaxis is now considered the gold standard for the treatment of severe haemophilia in childhood and adolescence, its use in adulthood is more controversial but here I argue that prophylaxis should be the gold standard in haemophilia treatment for life. […] The evidence that prophylaxis is effective is less strong in adults than it is in children but it is nevertheless convincing. […] For the majority of patients with standard severe haemophilia, however, adult prophylaxis should be the standard of care in the future as it will prevent bleeds, arthropathy and improve quality of life. […] Ultimately, as the authors of the editorial to accompany Manco-Johnsons paper in the New England Journal of Medicine state, „The dilemma in managing haemophilia is not whether to use prophylaxis or episodic treatment but how to manage prophylaxis such that the optimal, most cost effective treatment is provided”. […] I believe the statement applies equally well to the adult situation and for the majority of patients with severe haemophilia prophylactic treatment for life should be the new gold standard of care.

• #72 Advances in Hemophilia and the Role of Current and Emerging Prophylaxis

  https://www.ajmc.com/view/incorporating-emerging-innovation-hemophilia-ab-tailoring-prophylaxis-management-strategies-managed-care-environment-advances-emerging-prophylaxis

  Studies in adolescents and adults with hemophilia have shown significant benefits of secondary prophylaxis. […] Prophylaxis has been demonstrated to reduce the risk of life-threatening hemorrhages, including intracranial hemorrhage, compared with patients receiving only on-demand treatment. […] Although prophylaxis may decrease the frequency of bleeding episodes and decrease the progression of disease, it does not reverse established joint disease. […] Preventing painful, disabling, and costly joint arthropathy is a major goal of hemophilia management. Prophylactic treatment with clotting-factor concentrates is the standard of care for hemophilia A and B. […] Long-term prophylaxis is now widely used in severe hemophilia, even as clotting factors and replacement strategies continue to evolve. However, further research is needed to help clinicians develop individualized protocols and understand the impact of long-term use into adulthood.

• #73 Prophylaxis is able to reduce bleeds from severe hemophilia in older peopleEnvelope icon

  https://hemophilianewstoday.com/news/prophylaxis-reduce-bleeds-severe-hemophilia-older-people/

  Preventive therapy, or prophylaxis, in patients ages 50 and older with severe hemophilia type A and type B was effective at reducing bleeds, including those in target joints, and improving some aspects of their quality of life. […] Preventive or prophylactic replacement therapy is considered the gold standard treatment. It involves administering the clotting factor patients are missing on a routine basis as preventive measure against bleeds. […] With both types of hemophilia, prophylaxis reduced the overall annualized bleed rate (ABR) and the joint ABR, including in those with target joints at baseline, in patients ages 50 and older, the analysis showed. […] Quality of life, as assessed by the Hemophilia Quality of Life Questionnaire for Adults, was sustained in patients, ages 50 and older, with both types of hemophilia, with improvements seen in domains like physical health, and sports and leisure. The findings suggest prophylaxis “may enable older [people with hemophilia] to be more physically active,” the researchers wrote.

• #74 Advances in Hemophilia and the Role of Current and Emerging Prophylaxis

  https://www.ajmc.com/view/incorporating-emerging-innovation-hemophilia-ab-tailoring-prophylaxis-management-strategies-managed-care-environment-advances-emerging-prophylaxis

  Studies in adolescents and adults with hemophilia have shown significant benefits of secondary prophylaxis. […] Prophylaxis has been demonstrated to reduce the risk of life-threatening hemorrhages, including intracranial hemorrhage, compared with patients receiving only on-demand treatment. […] Although prophylaxis may decrease the frequency of bleeding episodes and decrease the progression of disease, it does not reverse established joint disease. […] Preventing painful, disabling, and costly joint arthropathy is a major goal of hemophilia management. Prophylactic treatment with clotting-factor concentrates is the standard of care for hemophilia A and B. […] Long-term prophylaxis is now widely used in severe hemophilia, even as clotting factors and replacement strategies continue to evolve. However, further research is needed to help clinicians develop individualized protocols and understand the impact of long-term use into adulthood.

• #75 Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A | Radhakrishnan | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/1346/870

  Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A […] Background: The real-world data on outcome of hemophilia A patients with inhibitors (HAI) is sparse, especially from developing countries. In a setting of inequitable healthcare opportunities for hemophilia patients, especially those with inhibitors, low-dose practices of emicizumab are emerging. In the present article, we describe our experience of managing HAI patients on low-dose emicizumab over a period of 56 months (from December 2019 to August 2024). […] Methods: The present study reports the response of patients with inhibitor-positive severe hemophilia A (HAI) and a high annual bleed rate to two-dose schedules of emicizumab prophylaxis. All patients with HAI were previously managed with on-demand bypassing agents (BPAs) before being shifted to emicizumab. Seven patients were treated on standard dose of 3 mg/kg weekly for 4 weeks followed by once in 2 weeks, whereas 25 patients were started on low dose of 3 mg/kg once in 4 weeks with or without loading as per clinical decision. […] Emicizumab prophylaxis even in lower doses is effective in preventing bleeds and improving joint outcome in HAI with pre-existing high bleed rate and arthropathy. This opens up an avenue for providing equity in healthcare delivery for HAI in low- and middle-income countries (LMICs) such as India.

• #76 Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A | Radhakrishnan | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/1346/870

  Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A […] Background: The real-world data on outcome of hemophilia A patients with inhibitors (HAI) is sparse, especially from developing countries. In a setting of inequitable healthcare opportunities for hemophilia patients, especially those with inhibitors, low-dose practices of emicizumab are emerging. In the present article, we describe our experience of managing HAI patients on low-dose emicizumab over a period of 56 months (from December 2019 to August 2024). […] Methods: The present study reports the response of patients with inhibitor-positive severe hemophilia A (HAI) and a high annual bleed rate to two-dose schedules of emicizumab prophylaxis. All patients with HAI were previously managed with on-demand bypassing agents (BPAs) before being shifted to emicizumab. Seven patients were treated on standard dose of 3 mg/kg weekly for 4 weeks followed by once in 2 weeks, whereas 25 patients were started on low dose of 3 mg/kg once in 4 weeks with or without loading as per clinical decision. […] Emicizumab prophylaxis even in lower doses is effective in preventing bleeds and improving joint outcome in HAI with pre-existing high bleed rate and arthropathy. This opens up an avenue for providing equity in healthcare delivery for HAI in low- and middle-income countries (LMICs) such as India.

• #77 Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A | Radhakrishnan | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/1346/870

  Patients with hemophilia need lifelong access to clotting factor concentrates, either as replacement for preventing or as episodic for treating bleeding. […] Emicizumab, a bispecific monoclonal antibody which binds to factor IXa and X and activates the latter has been approved for use in patients with inhibitor-positive hemophilia A (HAI). […] The study demonstrates how monoclonal agents that are currently licensed as well as those in the pipeline will be the game changer in hemophilia care, offering not only the advantage of subcutaneous products and less hospital visits, but also near-zero bleeds even at lower-dose range. It offsets not only the direct costs as has been demonstrated here, but also reduces the out-of-pocket expenses of the family by reducing hospital visits and medical infrastructure costs due to subcutaneous administration, lesser or probably no need for joint replacement surgeries in the future for these children. It also offers the promise of better school and job attendance and lesser chance of disability, as well as more employment opportunities for these patients.

• #78 Innovative Treatment Approaches in Routine Prophylaxis for Hemophilia A and B – Hematology Advisor

  https://www.hematologyadvisor.com/cch/hemophilia-a-b-prophylaxis-fviii-fix-marstacimab-emicizumab-concizumab/

  Methods that target tissue factor pathway inhibitor (TFPI) aim to enhance thrombin production and improve clotting by diminishing TFPI activity and thus facilitating more effective clot formation. […] Innovations are focusing on subcutaneous administration of nonfactor treatments, which offer longer half-lives and improve patient adherence through simplified treatment regimens. […] Clinical trials of marstacimab have shown a 92% reduction in bleeding rates and no thrombotic events. Potential Food and Drug Administration (FDA) approval is pending. […] As a leading nonfactor treatment for hemophilia A, emicizumab is widely prescribed and effective. […] No nonfactor treatment option is available for hemophilia B. Thus, patients with inhibitors are vulnerable to severe bleeding episodes.

• #79 Hemophilia: Treatment, Preventing Bleeding and Daily Care

  https://www.nationwidechildrens.org/conditions/hemophilia

  The key to best outcomes is to prevent and treat bleeding. You do this by making good choices of activities and wearing good safety equipment such as seat belts, bike helmets and sports safety gear. […] Here are some things you can do to help prevent bleeding: […] Be sure to tell the doctor, dentist or nurse your child has hemophilia. […] Be sure immunizations are given under the skin (subcutaneous) instead of into a muscle (intramuscular) and are given with a smaller needle. The staff should apply pressure and ice to the injection site for 5 to 10 minutes after the shot is given. This prevents bleeding. […] Do not let your child get intramuscular (IM) injections (shots). […] Never let a doctor or dentist do any procedure that involves opening the skin until you have talked with the Hemophilia team.

• #80 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  The main treatment for severe hemophilia involves replacing the clotting factor you need through a tube in a vein. […] This replacement therapy can be given to treat a bleeding episode in progress. It can also be given on a regular schedule at home to help prevent bleeding episodes. Some people receive continuous replacement therapy. […] To avoid excessive bleeding and protect your joints: […] Exercise regularly. Activities such as swimming, bicycle riding and walking can build muscles while protecting joints. […] Avoid certain pain medications. Drugs that can make bleeding worse include aspirin and ibuprofen (Advil, Motrin IB, others). Instead, use acetaminophen (Tylenol, others), which is a safer alternative for mild pain relief. […] Avoid blood-thinning medications. Medications that prevent blood from clotting include heparin, warfarin (Jantoven), clopidogrel (Plavix), prasugrel (Effient), ticagrelor (Brilinta), rivaroxaban (Xarelto), apixaban (Eliquis), edoxaban (Savaysa) and dabigatran (Pradaxa).

• #81 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  The main treatment for severe hemophilia involves replacing the clotting factor you need through a tube in a vein. […] This replacement therapy can be given to treat a bleeding episode in progress. It can also be given on a regular schedule at home to help prevent bleeding episodes. Some people receive continuous replacement therapy. […] To avoid excessive bleeding and protect your joints: […] Exercise regularly. Activities such as swimming, bicycle riding and walking can build muscles while protecting joints. […] Avoid certain pain medications. Drugs that can make bleeding worse include aspirin and ibuprofen (Advil, Motrin IB, others). Instead, use acetaminophen (Tylenol, others), which is a safer alternative for mild pain relief. […] Avoid blood-thinning medications. Medications that prevent blood from clotting include heparin, warfarin (Jantoven), clopidogrel (Plavix), prasugrel (Effient), ticagrelor (Brilinta), rivaroxaban (Xarelto), apixaban (Eliquis), edoxaban (Savaysa) and dabigatran (Pradaxa).

• #82 Prevention of Hemophila | Can Hemophilia Be Prevented| PatientsEngage

  https://www.patientsengage.com/conditions/hemophilia/prevention

  While Hemophilia cant be prevented, as it is a genetic disease. However, pre-conception screening in families with history of hemophilia, early diagnosis and appropriate management can significantly improve outcomes. Here are some preventive steps to limit the frequency and severity of bleeding in Hemophilia. […] Avoid exercises, sports or activities that place excessive strain on the joints to protect joints, as any minor trauma can lead to bleeding in patients. […] Regular medical checkups and tests for monitoring health status and follow-up of prophylactic treatment are very important to avoid complications. […] Low impact exercises like swimming or cycling to improve joint flexibility without risk of bleeding. […] Use protective devices to avoid injuries such as helmets during physical activities or driving to minimize the risk of traumatic injuries.

• #83 Treatment Approaches > Treatment and Care Options > HoG Handbook > Hemophilia of Georgia

  https://www.hog.org/handbook/section/3/treatment-approaches

  In the case of a target joint, where an individual with Hemophilia experiences repeated bleeding episodes in the same joint, a specific treatment approach may be employed to address the issue. […] To treat a target joint, the individual’s healthcare provider may prescribe a regimen where factor infusions are administered at regular intervals, typically every two to four days, for a few weeks or even longer. By doing so, the clotting factor levels in the person’s blood are elevated to a level that helps prevent further bleeding episodes in the affected joint. […] In addition to factor infusions, it is often recommended that individuals with Hemophilia engage in regular exercise to strengthen the muscles surrounding the affected joint. Strengthening these muscles can help provide additional support and stability to the joint, potentially reducing the likelihood of bleeding when treatments are discontinued or less frequent.

• #84 Hemophilia: Treatment, Preventing Bleeding and Daily Care

  https://www.nationwidechildrens.org/conditions/hemophilia

  People with bleeding disorders must talk to their doctor or nurse before taking medicine that can affect their platelets. These include aspirin, ibuprofen and naproxen. Always ask your hemophilia team before giving your child a medicine. […] Children with hemophilia should not play contact sports such as football, wrestling, boxing or hockey. If you have any questions about your child playing sports, be sure to talk with your hemophilia team.

• #85 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  The main treatment for severe hemophilia involves replacing the clotting factor you need through a tube in a vein. […] This replacement therapy can be given to treat a bleeding episode in progress. It can also be given on a regular schedule at home to help prevent bleeding episodes. Some people receive continuous replacement therapy. […] To avoid excessive bleeding and protect your joints: […] Exercise regularly. Activities such as swimming, bicycle riding and walking can build muscles while protecting joints. […] Avoid certain pain medications. Drugs that can make bleeding worse include aspirin and ibuprofen (Advil, Motrin IB, others). Instead, use acetaminophen (Tylenol, others), which is a safer alternative for mild pain relief. […] Avoid blood-thinning medications. Medications that prevent blood from clotting include heparin, warfarin (Jantoven), clopidogrel (Plavix), prasugrel (Effient), ticagrelor (Brilinta), rivaroxaban (Xarelto), apixaban (Eliquis), edoxaban (Savaysa) and dabigatran (Pradaxa).

• #86 Hemophilia: Treatment, Preventing Bleeding and Daily Care

  https://www.nationwidechildrens.org/conditions/hemophilia

  People with bleeding disorders must talk to their doctor or nurse before taking medicine that can affect their platelets. These include aspirin, ibuprofen and naproxen. Always ask your hemophilia team before giving your child a medicine. […] Children with hemophilia should not play contact sports such as football, wrestling, boxing or hockey. If you have any questions about your child playing sports, be sure to talk with your hemophilia team.

• #87 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  The main treatment for severe hemophilia involves replacing the clotting factor you need through a tube in a vein. […] This replacement therapy can be given to treat a bleeding episode in progress. It can also be given on a regular schedule at home to help prevent bleeding episodes. Some people receive continuous replacement therapy. […] To avoid excessive bleeding and protect your joints: […] Exercise regularly. Activities such as swimming, bicycle riding and walking can build muscles while protecting joints. […] Avoid certain pain medications. Drugs that can make bleeding worse include aspirin and ibuprofen (Advil, Motrin IB, others). Instead, use acetaminophen (Tylenol, others), which is a safer alternative for mild pain relief. […] Avoid blood-thinning medications. Medications that prevent blood from clotting include heparin, warfarin (Jantoven), clopidogrel (Plavix), prasugrel (Effient), ticagrelor (Brilinta), rivaroxaban (Xarelto), apixaban (Eliquis), edoxaban (Savaysa) and dabigatran (Pradaxa).

• #88 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  The main treatment for severe hemophilia involves replacing the clotting factor you need through a tube in a vein. […] This replacement therapy can be given to treat a bleeding episode in progress. It can also be given on a regular schedule at home to help prevent bleeding episodes. Some people receive continuous replacement therapy. […] To avoid excessive bleeding and protect your joints: […] Exercise regularly. Activities such as swimming, bicycle riding and walking can build muscles while protecting joints. […] Avoid certain pain medications. Drugs that can make bleeding worse include aspirin and ibuprofen (Advil, Motrin IB, others). Instead, use acetaminophen (Tylenol, others), which is a safer alternative for mild pain relief. […] Avoid blood-thinning medications. Medications that prevent blood from clotting include heparin, warfarin (Jantoven), clopidogrel (Plavix), prasugrel (Effient), ticagrelor (Brilinta), rivaroxaban (Xarelto), apixaban (Eliquis), edoxaban (Savaysa) and dabigatran (Pradaxa).

• #89 10 Interesting Facts about Hemophilia | thirdAGE

  https://thirdage.com/slideshow/10-interesting-facts-about-hemophilia/

  Globally, 75% of hemophiliacs do not get adequate treatment. […] Currently, hemophiliacs can be treated with an intravenous clotting factor, although this is not a cure and must be repeated as often as every other day. […] However, a research team in South Africa at the University of the Witwatersrand recently reported good results in a trial of subcutaneous (under the skin) injections of a protein called emicizumab, which is FDA approved for use in the U.S. […] This is still not a cure, but it is easier than intravenous injections and needs to be administered much less frequently. […] And on the horizon: experimental gene therapies have been yielding promising results in early trials, and may, in one treatment, be able to cure this condition. […] Although acute (short term) pain as well as chronic (long term) pain are common for those with hemophilia, aspirin is not recommended as a treatment option. […] According to Living with Hemophilia, aspirin and products containing aspirin can affect platelet function.

• #90 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  The main treatment for severe hemophilia involves replacing the clotting factor you need through a tube in a vein. […] This replacement therapy can be given to treat a bleeding episode in progress. It can also be given on a regular schedule at home to help prevent bleeding episodes. Some people receive continuous replacement therapy. […] To avoid excessive bleeding and protect your joints: […] Exercise regularly. Activities such as swimming, bicycle riding and walking can build muscles while protecting joints. […] Avoid certain pain medications. Drugs that can make bleeding worse include aspirin and ibuprofen (Advil, Motrin IB, others). Instead, use acetaminophen (Tylenol, others), which is a safer alternative for mild pain relief. […] Avoid blood-thinning medications. Medications that prevent blood from clotting include heparin, warfarin (Jantoven), clopidogrel (Plavix), prasugrel (Effient), ticagrelor (Brilinta), rivaroxaban (Xarelto), apixaban (Eliquis), edoxaban (Savaysa) and dabigatran (Pradaxa).

• #91 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  Practice good dental hygiene. The goal is to prevent tooth and gum disease, which can lead to excessive bleeding. […] Get vaccinations. People with hemophilia should receive recommended vaccinations at the appropriate ages, as well as hepatitis A and B. Requesting use of the smallest gauge needle and having pressure or ice applied for 3 to 5 minutes after the injection can reduce the risk of bleeding. […] Protect your child from injuries that could cause bleeding. Kneepads, elbow pads, helmets and safety belts all help prevent injuries from falls and other accidents. Keep your home free of furniture with sharp corners.

• #92 Prevention of Hemophila | Can Hemophilia Be Prevented| PatientsEngage

  https://www.patientsengage.com/conditions/hemophilia/prevention

  While Hemophilia cant be prevented, as it is a genetic disease. However, pre-conception screening in families with history of hemophilia, early diagnosis and appropriate management can significantly improve outcomes. Here are some preventive steps to limit the frequency and severity of bleeding in Hemophilia. […] Avoid exercises, sports or activities that place excessive strain on the joints to protect joints, as any minor trauma can lead to bleeding in patients. […] Regular medical checkups and tests for monitoring health status and follow-up of prophylactic treatment are very important to avoid complications. […] Low impact exercises like swimming or cycling to improve joint flexibility without risk of bleeding. […] Use protective devices to avoid injuries such as helmets during physical activities or driving to minimize the risk of traumatic injuries.

• #93 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  Practice good dental hygiene. The goal is to prevent tooth and gum disease, which can lead to excessive bleeding. […] Get vaccinations. People with hemophilia should receive recommended vaccinations at the appropriate ages, as well as hepatitis A and B. Requesting use of the smallest gauge needle and having pressure or ice applied for 3 to 5 minutes after the injection can reduce the risk of bleeding. […] Protect your child from injuries that could cause bleeding. Kneepads, elbow pads, helmets and safety belts all help prevent injuries from falls and other accidents. Keep your home free of furniture with sharp corners.

• #94 Hemophilia: Causes, types, symptoms, and treatment

  https://www.medicalnewstoday.com/articles/154880

  Hemophilia is an inherited bleeding disorder when the blood does not clot as it should. This can result in spontaneous bleeding and bruising after surgery or other injuries. […] Treatment focuses on replacing the missing protein and preventing complications. It involves giving or replacing the clotting factors that are too low or missing. […] Some patients will need regular replacement therapy in order to prevent bleeding. This is called prophylactic therapy. Doctors typically recommend regular treatment for people with severe forms of Hemophilia A. […] As preventive treatment, a person should follow the doctors recommendations regarding when to get injections of clotting factor VIII or IX. […] The CDC recommends regular testing for blood-borne infections, such as HIV and hepatitis. They also recommend getting vaccinations for hepatitis A and B. People with hemophilia who receive donated blood products may be at risk of contracting these conditions. […] To protect against injuries that can cause bleeding, a person can wear protective gear. They should also take extra precautions, such as consulting a physician, when participating in sports or high impact activities.

• #95 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  Practice good dental hygiene. The goal is to prevent tooth and gum disease, which can lead to excessive bleeding. […] Get vaccinations. People with hemophilia should receive recommended vaccinations at the appropriate ages, as well as hepatitis A and B. Requesting use of the smallest gauge needle and having pressure or ice applied for 3 to 5 minutes after the injection can reduce the risk of bleeding. […] Protect your child from injuries that could cause bleeding. Kneepads, elbow pads, helmets and safety belts all help prevent injuries from falls and other accidents. Keep your home free of furniture with sharp corners.

• #96 Hemophilia: Treatment, Preventing Bleeding and Daily Care

  https://www.nationwidechildrens.org/conditions/hemophilia

  The key to best outcomes is to prevent and treat bleeding. You do this by making good choices of activities and wearing good safety equipment such as seat belts, bike helmets and sports safety gear. […] Here are some things you can do to help prevent bleeding: […] Be sure to tell the doctor, dentist or nurse your child has hemophilia. […] Be sure immunizations are given under the skin (subcutaneous) instead of into a muscle (intramuscular) and are given with a smaller needle. The staff should apply pressure and ice to the injection site for 5 to 10 minutes after the shot is given. This prevents bleeding. […] Do not let your child get intramuscular (IM) injections (shots). […] Never let a doctor or dentist do any procedure that involves opening the skin until you have talked with the Hemophilia team.

• #97 Hemophilia – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

  Practice good dental hygiene. The goal is to prevent tooth and gum disease, which can lead to excessive bleeding. […] Get vaccinations. People with hemophilia should receive recommended vaccinations at the appropriate ages, as well as hepatitis A and B. Requesting use of the smallest gauge needle and having pressure or ice applied for 3 to 5 minutes after the injection can reduce the risk of bleeding. […] Protect your child from injuries that could cause bleeding. Kneepads, elbow pads, helmets and safety belts all help prevent injuries from falls and other accidents. Keep your home free of furniture with sharp corners.

• #98 Hemophilia: Causes, types, symptoms, and treatment

  https://www.medicalnewstoday.com/articles/154880

  Hemophilia is an inherited bleeding disorder when the blood does not clot as it should. This can result in spontaneous bleeding and bruising after surgery or other injuries. […] Treatment focuses on replacing the missing protein and preventing complications. It involves giving or replacing the clotting factors that are too low or missing. […] Some patients will need regular replacement therapy in order to prevent bleeding. This is called prophylactic therapy. Doctors typically recommend regular treatment for people with severe forms of Hemophilia A. […] As preventive treatment, a person should follow the doctors recommendations regarding when to get injections of clotting factor VIII or IX. […] The CDC recommends regular testing for blood-borne infections, such as HIV and hepatitis. They also recommend getting vaccinations for hepatitis A and B. People with hemophilia who receive donated blood products may be at risk of contracting these conditions. […] To protect against injuries that can cause bleeding, a person can wear protective gear. They should also take extra precautions, such as consulting a physician, when participating in sports or high impact activities.

• #99 Hemophilia: Treatment, Preventing Bleeding and Daily Care

  https://www.nationwidechildrens.org/conditions/hemophilia

  The key to best outcomes is to prevent and treat bleeding. You do this by making good choices of activities and wearing good safety equipment such as seat belts, bike helmets and sports safety gear. […] Here are some things you can do to help prevent bleeding: […] Be sure to tell the doctor, dentist or nurse your child has hemophilia. […] Be sure immunizations are given under the skin (subcutaneous) instead of into a muscle (intramuscular) and are given with a smaller needle. The staff should apply pressure and ice to the injection site for 5 to 10 minutes after the shot is given. This prevents bleeding. […] Do not let your child get intramuscular (IM) injections (shots). […] Never let a doctor or dentist do any procedure that involves opening the skin until you have talked with the Hemophilia team.

• #100 Hemophilia: Treatment, Preventing Bleeding and Daily Care

  https://www.nationwidechildrens.org/conditions/hemophilia

  The key to best outcomes is to prevent and treat bleeding. You do this by making good choices of activities and wearing good safety equipment such as seat belts, bike helmets and sports safety gear. […] Here are some things you can do to help prevent bleeding: […] Be sure to tell the doctor, dentist or nurse your child has hemophilia. […] Be sure immunizations are given under the skin (subcutaneous) instead of into a muscle (intramuscular) and are given with a smaller needle. The staff should apply pressure and ice to the injection site for 5 to 10 minutes after the shot is given. This prevents bleeding. […] Do not let your child get intramuscular (IM) injections (shots). […] Never let a doctor or dentist do any procedure that involves opening the skin until you have talked with the Hemophilia team.

• #101 Hemophilia A and B Prophylaxis Recommendations | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-267-masac-recommendation-concerning-prophylaxis-for-hemophilia-a-and-b-with-and-without-inhibitors

  Prevention of early life bleeding including mitigation of the risk of intracranial hemorrhage may be possible if emicizumab prophylaxis is implemented prior to 6 months of age. […] Adherence with prophylaxis should be monitored. Options for monitoring include self-report, electronic diaries, and pharmacy reports. […] Regular surveillance and utilization of a multidisciplinary team approach are critical to ensuring adherence to a mutually decided prophylactic regimen. […] Shared decision-making tools are available to help guide health care professionals and patients to guide decision-making about prophylaxis.

• #102 About Hemophilia | Hemophilia | CDC

  https://www.cdc.gov/hemophilia/about/index.html

  The standard way to treat both hemophilia A and B is to replace the missing blood clotting factor so that the blood can clot properly. One way to do this is by infusing (injecting into a vein) commercially prepared clotting factor concentrates. People with hemophilia can learn how to perform these infusions themselves so that they can stop bleeding episodes and, by performing the infusions on a regular basis (called prophylaxis), can even prevent most bleeding episodes. […] Good quality medical care from healthcare providers who know a lot about the disorder can help prevent some serious problems. This type of specialty care can be found at a comprehensive hemophilia treatment center (HTC). An HTC not only provides care to address all issues related to the disorder, but also provides health education that helps people with hemophilia stay healthy. Use the HTC directory to find a treatment center near you.

• #103 About Hemophilia | Hemophilia | CDC

  https://www.cdc.gov/hemophilia/about/index.html

  The standard way to treat both hemophilia A and B is to replace the missing blood clotting factor so that the blood can clot properly. One way to do this is by infusing (injecting into a vein) commercially prepared clotting factor concentrates. People with hemophilia can learn how to perform these infusions themselves so that they can stop bleeding episodes and, by performing the infusions on a regular basis (called prophylaxis), can even prevent most bleeding episodes. […] Good quality medical care from healthcare providers who know a lot about the disorder can help prevent some serious problems. This type of specialty care can be found at a comprehensive hemophilia treatment center (HTC). An HTC not only provides care to address all issues related to the disorder, but also provides health education that helps people with hemophilia stay healthy. Use the HTC directory to find a treatment center near you.

• #104 Hemophilia | UC Davis Hemostasis and Thrombosis Center | UC Davis Health

  https://health.ucdavis.edu/hemophilia/bleeding-disorders/hemophilia.html

  The best way to treat hemophilia is to replace the missing blood clotting factor so that the blood can clot properly. This is done by infusing (administering through a vein) commercially prepared factor concentrates. People with hemophilia can learn how to perform these infusions themselves so that they can stop bleeding episodes and by performing the infusions on a regular basis (called prophylaxis), can even prevent most bleeding episodes. […] Good quality medical care from doctors and nurses who know a lot about the disorder can help prevent some serious problems. Often the best choice for care is to visit a comprehensive Hemophilia Treatment Center (HTC), such as the UC Davis Hemostasis and Thrombosis Center. A HTC not only provides care to address all issues related to the disorder, but also provides health education that helps people with hemophilia stay healthy.

• #105 What Is Hemophilia?

  https://my.clevelandclinic.org/health/diseases/14083-hemophilia

  Healthcare providers treat hemophilia by replacing missing clotting factors. […] Healthcare providers treat hemophilia by boosting clotting factor levels or replacing missing clotting factors (replacement therapy). […] If you have severe hemophilia and frequent bleeding episodes, your healthcare provider may prescribe prophylactic factor infusions to prevent bleeding. […] No, they cant. If you have hemophilia and have children, your healthcare provider may recommend genetic testing so you and your children know whether they might pass hemophilia on to their children.

• #106 Hemophilia Review

  https://www.uspharmacist.com/article/hemophilia-revieww

  Another significant obstacle that hemophilia patients face is the development of inhibitors. […] Hemlibra, a factor IXa and factor X-directed antibody agent, is FDA approved for prophylaxis of hemophilia A patients who have inhibitors. […] The role of the pharmacist in the management of hemophilia continues to expand as new products continue to be developed. Pharmacists play an important role in drug selection, disease management, and education.

• #107 Hemophilia A & B Prophylaxis – Guidelines Side-By-Side – Guideline Central

  https://www.guidelinecentral.com/insights/hemophilia-side-by-side/

  For patients who are adherent to their prescribed prophylaxis regimen but still experience breakthrough bleeds: Recommends escalation of prophylaxis with measurement of trough levels and, if required, orthopedic interventions as appropriate. […] For patients with severe phenotype hemophilia A or B on prophylaxis: Recommend patients caregivers be taught to maintain timely and accurate records of bleeding episodes and treatment and be followed in hemophilia treatment centers. […] For patients with severe and moderately-severe hemophilia A without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events. […] For patients with severe hemophilia A with inhibitors: Suggest prophylaxis over episodic treatment of bleeding events. […] For patients with severe and moderately-severe hemophilia B without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events.

• #108 Prevention of Hemophila | Can Hemophilia Be Prevented| PatientsEngage

  https://www.patientsengage.com/conditions/hemophilia/prevention

  While Hemophilia cant be prevented, as it is a genetic disease. However, pre-conception screening in families with history of hemophilia, early diagnosis and appropriate management can significantly improve outcomes. Here are some preventive steps to limit the frequency and severity of bleeding in Hemophilia. […] Avoid exercises, sports or activities that place excessive strain on the joints to protect joints, as any minor trauma can lead to bleeding in patients. […] Regular medical checkups and tests for monitoring health status and follow-up of prophylactic treatment are very important to avoid complications. […] Low impact exercises like swimming or cycling to improve joint flexibility without risk of bleeding. […] Use protective devices to avoid injuries such as helmets during physical activities or driving to minimize the risk of traumatic injuries.

• #109 Hemophilia A & B Prophylaxis – Guidelines Side-By-Side – Guideline Central

  https://www.guidelinecentral.com/insights/hemophilia-side-by-side/

  For patients who are adherent to their prescribed prophylaxis regimen but still experience breakthrough bleeds: Recommends escalation of prophylaxis with measurement of trough levels and, if required, orthopedic interventions as appropriate. […] For patients with severe phenotype hemophilia A or B on prophylaxis: Recommend patients caregivers be taught to maintain timely and accurate records of bleeding episodes and treatment and be followed in hemophilia treatment centers. […] For patients with severe and moderately-severe hemophilia A without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events. […] For patients with severe hemophilia A with inhibitors: Suggest prophylaxis over episodic treatment of bleeding events. […] For patients with severe and moderately-severe hemophilia B without inhibitors: Recommend prophylaxis over episodic treatment of bleeding events.

• #110 Prevention of Hemophila | Can Hemophilia Be Prevented| PatientsEngage

  https://www.patientsengage.com/conditions/hemophilia/prevention

  Consume a balanced diet with vitamin K inclusion to support overall health and clotting function in the body. […] Maintain diary or record of bleeding episodes for further precautions during travelling or in emergency to seek medical care. It will be helpful for doctor to decide treatment plan. […] Prenatal testing and carrier screening to identify disease in the fetus […] Raise awareness about the disease in society to reduce stigma and improve access to care in the community.

• #111 Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review | Orphanet Journal of Rare Diseases | Full Text

  https://ojrd.biomedcentral.com/articles/10.1186/s13023-021-01919-w

  In countries like China, low-dose prophylaxis can help in preventing joint bleeds in the short term, but in the long term, standard-dose therapy has shown high adherence among young adults and better joint health, in turn improving the quality of life. […] Evidence suggests that prophylaxis with factor replacement drugs plays a significant role in reducing the number of bleeds per year and prevents joint damage when compared with on-demand treatment. […] The WFH recommends initiation of prophylaxis at any age to reduce hemarthrosis and slow down the progression of hemophilic arthropathy. […] British Society for Hematology (BSH) guidelines recommend that adolescents and adults with severe hemophilia should be encouraged to continue regular prophylaxis, and the frequency and dose should be adjusted according to their bleeding phenotype and individual pharmacokinetic (PK) data.

• #112 Hemophilia A and B Prophylaxis Recommendations | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-267-masac-recommendation-concerning-prophylaxis-for-hemophilia-a-and-b-with-and-without-inhibitors

  Prevention of early life bleeding including mitigation of the risk of intracranial hemorrhage may be possible if emicizumab prophylaxis is implemented prior to 6 months of age. […] Adherence with prophylaxis should be monitored. Options for monitoring include self-report, electronic diaries, and pharmacy reports. […] Regular surveillance and utilization of a multidisciplinary team approach are critical to ensuring adherence to a mutually decided prophylactic regimen. […] Shared decision-making tools are available to help guide health care professionals and patients to guide decision-making about prophylaxis.

• #113 Considerations for Prophylaxis in Patients With Hemophilia A

  https://www.pharmacytimes.com/view/considerations-for-prophylaxis-in-patients-with-hemophilia-a

  Yes, in very individualized cases, absolutely, we would consider discontinuing prophylaxis if the burdens outweigh the benefits that are being realized in the individual. […] Its never going to be normal, but were trying to get them so they can participate in life and physical activities some sports, obviously noncontact. […] Patient preference is certainly important in the selection of treatment and should be considered in decision-making, so we can help make the optimal treatment choice for not only favorable outcomes but sustainability in terms of adherence. […] In terms of some patient preferences that we see at the pharmacy level, aside from cost, 1 of the concerns is for developing inhibitors against Factor VIII replacement products.

• #114 Considerations for Prophylaxis in Patients With Hemophilia A

  https://www.pharmacytimes.com/view/considerations-for-prophylaxis-in-patients-with-hemophilia-a

  Yes, in very individualized cases, absolutely, we would consider discontinuing prophylaxis if the burdens outweigh the benefits that are being realized in the individual. […] Its never going to be normal, but were trying to get them so they can participate in life and physical activities some sports, obviously noncontact. […] Patient preference is certainly important in the selection of treatment and should be considered in decision-making, so we can help make the optimal treatment choice for not only favorable outcomes but sustainability in terms of adherence. […] In terms of some patient preferences that we see at the pharmacy level, aside from cost, 1 of the concerns is for developing inhibitors against Factor VIII replacement products.

• #115 Considerations for Prophylaxis in Patients With Hemophilia A

  https://www.pharmacytimes.com/view/considerations-for-prophylaxis-in-patients-with-hemophilia-a

  Yes, in very individualized cases, absolutely, we would consider discontinuing prophylaxis if the burdens outweigh the benefits that are being realized in the individual. […] Its never going to be normal, but were trying to get them so they can participate in life and physical activities some sports, obviously noncontact. […] Patient preference is certainly important in the selection of treatment and should be considered in decision-making, so we can help make the optimal treatment choice for not only favorable outcomes but sustainability in terms of adherence. […] In terms of some patient preferences that we see at the pharmacy level, aside from cost, 1 of the concerns is for developing inhibitors against Factor VIII replacement products.

• #116 Unmet Needs in Hemophilia: Prophylaxis, Bleeding, Quality of Life

  https://www.ajmc.com/view/unmet-needs-in-hemophilia-prophylaxis-bleeding-quality-of-life

  Over one-third of hemophilia patients experience bleeding episodes and joint damage despite prophylactic therapies, indicating a need for more effective treatments. […] There is a persistent unmet need for more effective therapies for hemophilia. […] Despite these therapies, many patients experienced breakthrough bleeding and moderate to severe joint problems. […] These findings show a continued unmet need for more effective therapies that not only focus on preventing bleeding but also improve joint health and address the psychological aspects of living with the disease. […] The authors emphasized that it is essential for stakeholders to prioritize innovative therapeutic strategies and holistic care models to enhance the overall well-being of individuals with hemophilia.

• #117 Hemophilia Review

  https://www.uspharmacist.com/article/hemophilia-revieww

  Hemophilia, an X-linked genetic disorder, is one of the most common serious congenital clotting-factor deficiencies. […] In order to prevent hemophilic arthropathy and other spontaneous bleeds, each week these patients receive prophylaxis consisting of multiple injections of concentrated amounts of the clotting factor they are deficient in. There are two types of hemophilia prophylaxis: primary prophylaxis, occurring before or at the start of the initial interarticular bleed, and secondary prophylaxis, administered once hemophilic arthropathy has progressed. […] Despite general success in the vast majority of patients with hemophilia, prophylaxis with conventional factor concentrates still faces some significant obstacles, including a short half-life. […] Novel technologies include pegylation, fusion-protein technology, and single-chain technology.

• #118 Unmet Needs in Hemophilia: Prophylaxis, Bleeding, Quality of Life

  https://www.ajmc.com/view/unmet-needs-in-hemophilia-prophylaxis-bleeding-quality-of-life

  Over one-third of hemophilia patients experience bleeding episodes and joint damage despite prophylactic therapies, indicating a need for more effective treatments. […] There is a persistent unmet need for more effective therapies for hemophilia. […] Despite these therapies, many patients experienced breakthrough bleeding and moderate to severe joint problems. […] These findings show a continued unmet need for more effective therapies that not only focus on preventing bleeding but also improve joint health and address the psychological aspects of living with the disease. […] The authors emphasized that it is essential for stakeholders to prioritize innovative therapeutic strategies and holistic care models to enhance the overall well-being of individuals with hemophilia.

• #119

  https://link.springer.com/article/10.1007/s12288-019-01147-0

  Several themes related to Hemophilia care like inhibitor development, tolerance, pharmacokinetics of CFCs and costbenefit analysis of different prophylaxis regimens are currently understood poorly. These distinct elements are highly relevant to assess the actual benefits of LDP regimen in a global scale.

• #120 Past, present & future of hemophilia treatment: from prophylaxis to gene therapies | VJHemOnc

  https://www.vjhemonc.com/feature/past-present-future-of-hemophilia-treatment-from-prophylaxis-to-gene-therapies/

  Emicizumab was approved by the FDA for patients with hemophilia A with and without inhibitors in 2018. This was based on the Phase III HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160) trials, where treatment reduced the ABR of patients with and without inhibitors compared to no prophylaxis. […] The novel approaches discussed here increase treatment options for patients with hemophilia who develop inhibitors from factor replacement therapy, or who cannot afford or manage weekly infusions. Novel non-replacement therapies are effective for patients with hemophilia A and B regardless of their inhibitor status, and the recently-approved AAV-based gene therapies offer the potential for a one-time treatment for hemophilia A and B. […] While there are still hurdles regarding the treatment and financial burden on patients, the therapeutic landscape for hemophilia is constantly evolving and improving.

• #121 10 Interesting Facts about Hemophilia | thirdAGE

  https://thirdage.com/slideshow/10-interesting-facts-about-hemophilia/

  Globally, 75% of hemophiliacs do not get adequate treatment. […] Currently, hemophiliacs can be treated with an intravenous clotting factor, although this is not a cure and must be repeated as often as every other day. […] However, a research team in South Africa at the University of the Witwatersrand recently reported good results in a trial of subcutaneous (under the skin) injections of a protein called emicizumab, which is FDA approved for use in the U.S. […] This is still not a cure, but it is easier than intravenous injections and needs to be administered much less frequently. […] And on the horizon: experimental gene therapies have been yielding promising results in early trials, and may, in one treatment, be able to cure this condition. […] Although acute (short term) pain as well as chronic (long term) pain are common for those with hemophilia, aspirin is not recommended as a treatment option. […] According to Living with Hemophilia, aspirin and products containing aspirin can affect platelet function.

• #122 Innovative Treatment Approaches in Routine Prophylaxis for Hemophilia A and B – Hematology Advisor

  https://www.hematologyadvisor.com/cch/hemophilia-a-b-prophylaxis-fviii-fix-marstacimab-emicizumab-concizumab/

  Recent advances in hemophilia treatment have revealed promising strategies for improving patient outcomes while minimizing the therapeutic burden. […] Marstacimab has shown considerable promise in clinical trials, achieving statistically significant and clinically meaningful reductions in annualized bleeding rates compared with conventional prophylaxis and on-demand intravenous treatments. […] Another promising therapy is concizumab, a monoclonal antibody that also targets TFPI to enhance thrombin generation. […] Innovative nonfactor treatments are undergoing exploration for subcutaneous administration, and they demonstrate significantly longer half-lives than current factor therapies. […] Unmet needs include the development of subcutaneous and self-administered options to improve patient access and adherence, along with the management of side effects such as injection site reactions.

• #123 Pharmacokinetic model-based assessment of factor IX prophylaxis treatment regimens in severe hemophilia B | Scientific Reports

  https://www.nature.com/articles/s41598-024-70784-x

  There is a need for resources which can adequately compare the optimal posology (i.e., the dosing schedule to reach a predefined target steady state FIX activity trough level using the lowest possible dose and administration frequency) of FIX products in patients with severe HB. […] The model estimates the ability of different FIX therapies to achieve target trough levels and the dosing schedule necessary to achieve this. […] This model also shows that some FIX therapies are either unable to reach certain targets or can do so only in a small proportion of patients. […] This PK model-based assessment may help reduce treatment burden, resource utilization and costs, while maintaining or lowering bleeding risk in people with severe HB by providing estimated optimal posologies for FIX therapies. […] The posology considered in this analysis is based on existing SmPCs, providing clinically relevant scenarios for supporting treatment decisions when used alongside clinical patient evaluation, including individual bleeding phenotype and both peak and trough FIX activity levels.

• #124 Optimization of prophylaxis for hemophilia A | PLOS One

  https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0192783

  The first step in identifying optimal prophylaxis regimens is to write equations that describe the time course of changes in plasma factor VIII concentrations (the pharmacokinetic trajectory) produced by any prophylaxis regimen. […] The grid search provides further information about how the prophylaxis regimen influences the lowest factor VIII concentration. […] The optimal prophylaxis regimen for this very active child involves injections of 30, 30 and 30 IU/kg administered on Wednesday at 3:00 pm, Saturday at 9:00 am and Sunday at 9:00 am. […] The implication is that, for this child, any regimen with a total dose of 90 IU/kg administered in up to three injections per week confers a similar risk of bleeds. […] The authors are developing a web-based calculator, called MOrPH, which will conduct the calculations described here.

• #125 Individualized prophylaxis for optimizing hemophilia care: can we apply this to both developed and developing nations? | Thrombosis Journal | Full Text

  https://thrombosisjournal.biomedcentral.com/articles/10.1186/s12959-016-0096-y

  Prophylaxis is considered optimal care for hemophilia patients to prevent bleeding and to preserve joint function thereby improving quality of life (QoL). […] Prophylaxis can be further individualized to improve outcomes and cost effectiveness. […] Prophylaxis in economically constrained nations is limited by the ill-affordability of clotting factor concentrates. […] However, at least 5 studies on children and adults from Thailand, China and India have shown superiority of low dose (~510 IU kg1 2-3 per week) prophylaxis over episodic treatment in terms of bleed reduction, and quality of life, with improved physical activity, independent functioning, school attendance and community participation. […] Prophylaxis must be individualized to include affordability as a major determinant in order to optimize low-dose regimens that have demonstrated superiority compared to episodic treatment. […] Prophylaxis has significantly changed the lives of many hemophilia patients. […] Low and very-low dose prophylaxis and individualizing it to what is affordable for a given patient or nation, may bridge this gap created by global economic disparity.

• #126 Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A | Radhakrishnan | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/1346/870

  Freedom From Bleeds With Low-Dose Emicizumab Prophylaxis in Inhibitor-Positive Hemophilia A […] Background: The real-world data on outcome of hemophilia A patients with inhibitors (HAI) is sparse, especially from developing countries. In a setting of inequitable healthcare opportunities for hemophilia patients, especially those with inhibitors, low-dose practices of emicizumab are emerging. In the present article, we describe our experience of managing HAI patients on low-dose emicizumab over a period of 56 months (from December 2019 to August 2024). […] Methods: The present study reports the response of patients with inhibitor-positive severe hemophilia A (HAI) and a high annual bleed rate to two-dose schedules of emicizumab prophylaxis. All patients with HAI were previously managed with on-demand bypassing agents (BPAs) before being shifted to emicizumab. Seven patients were treated on standard dose of 3 mg/kg weekly for 4 weeks followed by once in 2 weeks, whereas 25 patients were started on low dose of 3 mg/kg once in 4 weeks with or without loading as per clinical decision. […] Emicizumab prophylaxis even in lower doses is effective in preventing bleeds and improving joint outcome in HAI with pre-existing high bleed rate and arthropathy. This opens up an avenue for providing equity in healthcare delivery for HAI in low- and middle-income countries (LMICs) such as India.

• #127 Prophylaxis in haemophilia should be life-long

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3320774/

  Prophylaxis in haemophilia should be life-long. […] Although prophylaxis is now considered the gold standard for the treatment of severe haemophilia in childhood and adolescence, its use in adulthood is more controversial but here I argue that prophylaxis should be the gold standard in haemophilia treatment for life. […] The evidence that prophylaxis is effective is less strong in adults than it is in children but it is nevertheless convincing. […] For the majority of patients with standard severe haemophilia, however, adult prophylaxis should be the standard of care in the future as it will prevent bleeds, arthropathy and improve quality of life. […] Ultimately, as the authors of the editorial to accompany Manco-Johnsons paper in the New England Journal of Medicine state, „The dilemma in managing haemophilia is not whether to use prophylaxis or episodic treatment but how to manage prophylaxis such that the optimal, most cost effective treatment is provided”. […] I believe the statement applies equally well to the adult situation and for the majority of patients with severe haemophilia prophylactic treatment for life should be the new gold standard of care.

• #128 Hemophilia A & B Prophylaxis – Guidelines Side-By-Side – Guideline Central

  https://www.guidelinecentral.com/insights/hemophilia-side-by-side/

  Hemophilia A and B, the most common severe hereditary bleeding disorders, arise from deficiencies in factor VIII and factor IX, respectively. Apart from treating acute bleeding, prophylaxis is a key strategy in hemophilia management. Prophylactic treatment significantly reduces the frequency of hemarthroses, thereby mitigating hemophilic arthropathy and the need for corrective joint surgeries. It also lowers the incidence of cerebral and muscle bleeds, decreases hospitalizations, and enhances patients quality of life by reducing time off work and less frequent monitoring. […] For patients with hemophilia A or B with a severe phenotype: Strongly recommend patients be on prophylaxis sufficient to prevent bleeds at all times in an individualized approach. […] For patients with severe phenotype hemophilia A or B, especially children: Recommend regular long-term prophylaxis as the standard of care to prevent hemarthrosis and other spontaneous and breakthrough bleeding, maintain musculoskeletal health, and promote quality of life.

• #129 Prophylaxis in haemophilia should be life-long

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3320774/

  Prophylaxis in haemophilia should be life-long. […] Although prophylaxis is now considered the gold standard for the treatment of severe haemophilia in childhood and adolescence, its use in adulthood is more controversial but here I argue that prophylaxis should be the gold standard in haemophilia treatment for life. […] The evidence that prophylaxis is effective is less strong in adults than it is in children but it is nevertheless convincing. […] For the majority of patients with standard severe haemophilia, however, adult prophylaxis should be the standard of care in the future as it will prevent bleeds, arthropathy and improve quality of life. […] Ultimately, as the authors of the editorial to accompany Manco-Johnsons paper in the New England Journal of Medicine state, „The dilemma in managing haemophilia is not whether to use prophylaxis or episodic treatment but how to manage prophylaxis such that the optimal, most cost effective treatment is provided”. […] I believe the statement applies equally well to the adult situation and for the majority of patients with severe haemophilia prophylactic treatment for life should be the new gold standard of care.

• #130 Hemophilia A and B Prophylaxis Recommendations | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-267-masac-recommendation-concerning-prophylaxis-for-hemophilia-a-and-b-with-and-without-inhibitors

  In view of the demonstrated benefits of prophylaxis begun at a young age in persons with hemophilia A or B (PwH), MASAC recommends that prophylaxis be considered standard of care therapy for individuals with severe hemophilia A or B (factor VIII or factor IX 1%) including those with inhibitors. Prophylactic therapy may also be considered for PwH with moderate and mild hemophilia with a severe phenotype. Prophylactic therapy should be instituted early (prior to the onset of frequent bleeding). […] Prophylaxis should be initiated at an early age, ideally before age 3 years and prior to the second joint bleed; prophylaxis may be considered within the first six months of life to reduce occurrence of intracranial hemorrhage. […] The goals for emicizumab prophylaxis remain like that of factor prophylaxis in that a regular administration of a prophylactic hemostatic agent should be considered as early as feasible to prevent the long-term complications of musculoskeletal bleeding and allow the patient to lead a physically active life approaching the quality of life of an unaffected individual.

• #131 NUWIQ® Personalized Prophylaxis | Optimizing Hemophilia A Treatmentnuwiq-logo

  https://nuwiqusa.com/hcp/dosing/personalized-prophylaxis/

  Optimizing FVIII therapy can be challenging—balancing dose and dosing frequency to prevent or reduce bleeding, while minimizing side effects, increasing convenience, and controlling overall treatment costs. Every hemophilia patient is unique, so a “one size fits all” approach to FVIII prophylaxis is not ideal. […] Personalized prophylaxis, whereby FVIII dose and dosing frequency is tailored to a patient’s individual pharmacokinetic data, has the potential to optimize care and enable fewer infusions. […] The NuPreviq clinical study showed that personalized prophylaxis with NUWIQ is effective. […] Personalized prophylaxis has the potential to optimize care and enable fewer infusions. […] Personalized Prophylaxis with NUWIQ Enabled the Majority of Patients to Extend Their Dosing Interval to Twice Weekly or Less.

• #132 Past, present & future of hemophilia treatment: from prophylaxis to gene therapies | VJHemOnc

  https://www.vjhemonc.com/feature/past-present-future-of-hemophilia-treatment-from-prophylaxis-to-gene-therapies/

  Emicizumab was approved by the FDA for patients with hemophilia A with and without inhibitors in 2018. This was based on the Phase III HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160) trials, where treatment reduced the ABR of patients with and without inhibitors compared to no prophylaxis. […] The novel approaches discussed here increase treatment options for patients with hemophilia who develop inhibitors from factor replacement therapy, or who cannot afford or manage weekly infusions. Novel non-replacement therapies are effective for patients with hemophilia A and B regardless of their inhibitor status, and the recently-approved AAV-based gene therapies offer the potential for a one-time treatment for hemophilia A and B. […] While there are still hurdles regarding the treatment and financial burden on patients, the therapeutic landscape for hemophilia is constantly evolving and improving.

• #133 Hemophilia A and B Prophylaxis Recommendations | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-267-masac-recommendation-concerning-prophylaxis-for-hemophilia-a-and-b-with-and-without-inhibitors

  In view of the demonstrated benefits of prophylaxis begun at a young age in persons with hemophilia A or B (PwH), MASAC recommends that prophylaxis be considered standard of care therapy for individuals with severe hemophilia A or B (factor VIII or factor IX 1%) including those with inhibitors. Prophylactic therapy may also be considered for PwH with moderate and mild hemophilia with a severe phenotype. Prophylactic therapy should be instituted early (prior to the onset of frequent bleeding). […] Prophylaxis should be initiated at an early age, ideally before age 3 years and prior to the second joint bleed; prophylaxis may be considered within the first six months of life to reduce occurrence of intracranial hemorrhage. […] The goals for emicizumab prophylaxis remain like that of factor prophylaxis in that a regular administration of a prophylactic hemostatic agent should be considered as early as feasible to prevent the long-term complications of musculoskeletal bleeding and allow the patient to lead a physically active life approaching the quality of life of an unaffected individual.

• #134 Primary prophylaxis in children with haemophilia

  https://pmc.ncbi.nlm.nih.gov/articles/PMC2652217/

  On the whole, severe haemophiliacs on prophylaxis and their families have a better quality of life than patients who receive on-demand treatment. […] The definitions of prophylaxis, therefore, reflect a wide spectrum of clinical conditions and objectives of the treatment, from the prevention of severe or life-threatening bleeds to the absence of arthropathy enabling patients to live a substantially normal life, without overprotection. […] In a congenital, chronic disease such as haemophilia, for which a definitive cure is still not available, the latter perspective is an important and reliable goal, achieved by primary prophylaxis. […] The key point is, therefore, likely to be the number of joint bleeds before the start of prophylaxis, as also shown in the Dutch experience with lower dose regimens, tailored to the patients bleeding tendency.

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