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. […] In practice, the frequency and severity of bleeding in an individual can only be defined clinically and it is, therefore, important that all patients keep good documentation of the treatments they self-administer. 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”. […] for the majority of patients with severe haemophilia prophylactic treatment for life should be the new gold standard of care.

• #1 Prophylaxis | The Haemophilia Society

  https://haemophilia.org.uk/bleeding-disorders/treatment-types/prophylaxis/

  Prophylaxis is the name given to regular treatment to prevent bleeds. For someone with haemophilia, this is treatment, typically 3 times a week, which helps the blood clot and minimises the likelihood of bleeds and long-term joint damage. […] Prophylaxis is hugely important for people living with haemophilia. Severe haemophiliacs who experience spontaneous bleeds into joints may sustain long-term damage such as arthritis, with certain joints becoming more vulnerable to target joints.

• #1 Prophylaxis is the new standard of care in patients with haemophilia

  http://www.scielo.org.za/scielo.php?script=sci_arttext&pid=S0256-95742022000600007

  Prophylaxis is the new standard of care in patients with haemophilia. […] Randomised controlled clinical trial evidence on prophylaxis as optimal care for patients with haemophilia was generated more than a decade ago. […] The recently introduced non-factor therapies in haemophilia care have addressed many of these barriers. […] Prophylaxis is the regular administration of a haemostatic agent or agents that safely, effectively and conveniently prevent bleeding while allowing persons with haemophilia to live active lives. […] The latest World Federation of Haemophilia (WFH) 2020 haemophilia treatment guideline has recommended that prophylaxis is the new global standard of care for all patients with haemophilia. […] Prophylaxis is therefore the recommended standard of care, even in resource-constrained settings.

• #1 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. […] Prophylaxis is not universally accepted, with only about half the children with hemophilia A receiving this treatment modality in the United States. Reasons cited for the lack of acceptance include need for venous access, factor availability, repeated venipunctures, and cost, among others. Research questions that remain unanswered include when to initiate and stop infusions, dosing, and dose schedule. […] 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.

• #1 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

  Prophylactic treatment with clotting-factor concentrates has been shown to prevent hemophilic arthropathy and is, therefore, the standard of care for hemophilia A and B. […] Early initiation with primary prophylaxis is ideal, but secondary prophylaxis in adolescents and adults has also demonstrated significant success. […] Because the standard of care includes prophylaxis with factor-concentrate replacement in order to prevent joint damage in patients with hemophilia, prophylaxis is now more common and needs to be addressed in all clinical settings, including managed care. […] The optimal regimen for initiating prophylaxis, duration of treatment, and dosing regimens continue to be studied. […] Prophylaxis is the administration of replacement clotting-factor concentrates in anticipation of bleeding or to prevent it.

• #1 Treating and Managing a Bleed | The Haemophilia Society

  https://haemophilia.org.uk/bleeding-disorders/haemophilia-a-and-b/treating-managing-a-bleed/

  Bleeding must be treated as soon as possible. Prompt treatment helps relieve pain quickly, shortens recovery time and reduces the chance of permanent damage. […] Prophylaxis stops the bleeding that occurs without any obvious injury, often called spontaneous bleeding. It also reduces the risk of bleeding from minor injuries. Research has shown that prophylaxis gives children the best chance to reach adulthood without damage to their joints. How often the infusions need to be given is decided for the individual but is typically every other day for haemophilia A and 2-3 times a week for haemophilia B because factor IX lasts a bit longer in the blood. […] Treatment with regular infusions of clotting factor aims to prevent the long-term damage caused by bleeding into joints and muscles.

• #1

  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 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.

• #1 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

  The difference between the two treatments is stark. On-demand patients could average 20-30 bleeds yearly, miss half their work or school days and face certain joint damage. Those on prophylaxis have an average of up to two bleeds yearly, attend 90 per cent of work/school, and maintain healthy joints. While prophylaxis costs more upfront, its actually cheaper in the long term when you factor in emergency care costs and lost productivity. […] Continuing with crisis care makes no economic sense. 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. India needs three urgent changes. First, include prophylaxis in all state health schemes. Second, negotiate bulk discounts to halve clotting factor costs. Third, train doctors and health workers to spot early signs, such as excessive bruising in toddlers. […] Haemophilia isnt just stealing health but also Indias human potential. We can keep paying the endless costs of disability and lost productivity or invest in solutions that give thousands of Indians their futures back. The choice is clear and the time to act is now.

• #1 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. […] Prophylaxis may be initiated with a dose-escalation strategy, starting at once weekly dosing, and then increasing as needed based on bleed frequency.

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

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

  Children who begin primary or secondary prophylaxis at an early age can often avoid serious joint bleeds and their complications, such as hemophilic arthropathy, which is when the joint is permanently damaged and causes disability. […] 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.

• #1 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.

• #1 JLE – Hématologie – Haemophilia prophylaxis in paediatrics

  https://www.jle.com/en/revues/hma/e-docs/prophylaxie_de_lhemophilie_en_pediatrie_352439/article.phtml

  The aim of paediatric haemophilia prophylaxis is to prevent recurrent bleeding and its complications. Prophylaxis, introduced early (ideally before the age of 2), aims to prevent irreversible joint damage and other serious complications such as intracranial haemorrhage. This may include regular injections of coagulation factors or, more recently, the use of emicizumab, a more practical subcutaneous treatment that transforms severe forms into minor ones. […] Regular clinical and biological monitoring is crucial for assessing efficacy, adapting treatments and preventing complications. This strategy significantly improves patients quality of life.

• #1 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.

• #1 Benefit of Prophylaxis for Adult Haemophilia A Patients – BJH

  https://www.bjh.be/benefit-of-prophylaxis-for-adult-haemophilia-a-patients/

  Prophylaxis with factor VIII (FVIII) concentrates in children with haemophilia A is current standard of care. However, the benefit of prophylactic treatment for adult haemophilia A patients is not commonly accepted. A new study shows that the use of prophylaxis is also beneficial in this group of patients. […] Prophylaxis had the greatest effect on the ABRs of patients of any age with severe or non-severe HA. […] In conclusion, prophylaxis resulted in lower ABR than on-demand treatment. Patients switching to prophylaxis benefitted the most, irrespective of age or disease severity. Prophylactic treatment is beneficial for patients of any age, including elderly patients, with severe or non-severe hemophilia A.

• #1 The current approaches to prophylaxis in hemophilia and tailoring treatment to the individual | VJHemOnc

  https://www.vjhemonc.com/video/5iiyswaw-vu-the-current-approaches-to-prophylaxis-in-hemophilia-and-tailoring-treatment-to-the-individual/

  The current approaches to prophylaxis in hemophilia highlight the importance of individualized treatment plans based on the type of hemophilia, age, and patient preference. […] Prophylaxis is crucial for patients with severe hemophilia, but novel approaches are also appropriate for patients with moderate and mild disease who wish to receive prophylaxis for various personal reasons. […] Prophylaxis is best for patients with severe hemophilia to suppress or prevent bleeding episodes. […] Many patients with moderate hemophilia may also have a more severe bleeding phenotype and should be considered for prophylaxis. […] There are a variety of things that we can utilize for prophylaxis. These methods for prophylaxis, again, don’t just apply to patients with severe disease. They also apply to patients with moderate disease and some patients with mild disease who participate in very strenuous activities or have individual life goals that require them to have higher levels.

• #1 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.

• #1 Exploring haemophilia prophylaxis – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-226-june-2024/exploring-haemophilia-prophylaxis/

  I see this evolution of haemophilia treatment as being corelated to prevention as the core of health care. […] Dr Maria Elisa Mancuso, a haematologist from Italy, suggested based on her research findings that prophylaxis treatment should maintain a high factor VIII (8) trough level at 40% or above to achieve zero bleeds and that prophylaxis treatment should also be available for mild haemophilia patients. […] Dr Carcao suggested that prophylaxis treatment should be started as early as possible to achieve joint health preservation and minimise bleeds. […] However, he noted that prophylaxis treatment in haemophilia is expensive and around 80% of the world has very little or zero access to the products. […] Consequently, although low dose prophylaxis tailored to individual needs and affordability are not enough, they are much better than no prophylaxis.

• #1 Hemophilia Review

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

  Both moderate-hemophilia and severe-hemophilia patients are extremely likely to experience a type of joint damage known as hemophilic arthropathy. 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. Historically, hemophilia prophylaxis was given in standardized weight-based doses; all patients, regardless of variability in tendency to bleed, pharmacokinetics, and hemophilic arthropathy, received three or four doses of 20 U/dL to 40 U/dL every week. However, personalized prophylaxis has since become a standard of prophylactic treatment. Patients express satisfaction when their routine prophylactic treatments are adjusted in accordance with their activity level, bleed history, and lifestyle demands.

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

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

  Both standard half-life FIX and extended half-life recombinant FIX (EHL rFIX) are effective treatment options for prophylaxis and can be used to offer adequate hemostatic cover for bleeds, surgery, and invasive procedures; however, when using EHLs, consider product-specific laboratory requirements for monitoring. […] Adapt the dose and frequency of FIX prophylaxis treatment to the clinical phenotype (eg, bleed rates) and lifestyle considerations and not to plasma trough levels exclusively.

• #1 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.

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

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

  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. The HAVEN 3 trial demonstrated that emicizumab prophylaxis is also effective in patients without inhibitors.

• #1 FDA approves siRNA drug for haemophilia prophylaxis

  https://www.nature.com/articles/d41573-025-00066-2

  Sanofi and Alnylams fitusiran (Qfitlia) has secured FDA approval for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients 12 years of age and older with haemophilia A or B, with or without factor VIII or IX inhibitors. […] The antithrombin-targeted siRNA can be dosed once every two months, potentially giving it a convenience edge over anticoagulant antibodies and factor VIII and factor IX products that are dosed more frequently.

• #1 Treatment of Hemophilia | Hemophilia | CDC

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

  Healthcare providers typically prescribe treatment products for episodic care or prophylactic care. […] Prophylactic care is used to prevent bleeding episodes from occurring. […] People with hemophilia can learn how to perform infusions themselves so that they can stop bleeding episodes and, by performing the infusions on a regular basis, can even prevent most bleeding episodes. […] This treatment product can be given by injection under the skin. […] Patients who use Hemlibra for prophylaxis and use clotting factor concentrates to treat breakthrough bleeds can still develop an inhibitor. […] Roctavian helps prevent bleeding. […] Hemgenix helps prevent bleeding.

• #1 Exploring haemophilia prophylaxis – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-226-june-2024/exploring-haemophilia-prophylaxis/

  Non-factor treatment is an option for personalised prophylaxis treatment, but raises questions such as the risk of thrombosis, cost and how individuals will handle the variety of medications when experiencing a bleed. […] Dr Veena Selvaratnam, a haematologist from Malaysia, presented the results on her low dose emicizumab studies, showing that low dose emicizumab resulted in zero bleeds in 9 out of 10 patients over 37- 293 weeks, and their QoL (quality of life) score improved significantly.

• #1 Emicizumab prophylaxis effectively prevents bleeding in patients with acquired haemophilia A – BJH

  https://www.bjh.be/emicizumab-prophylaxis-effectively-prevents-bleeding-in-patients-with-acquired-haemophilia-a/

  Emicizumab prophylaxis effectively prevents bleeding in patients with acquired haemophilia A. The findings, recently published in The Lancet Haematology, suggest that prophylaxis with emicizumab offers an effective protection against bleeding and show that the initiation of immunosuppressive therapy can be delayed while receiving this treatment. […] This study suggests that emicizumab prophylaxis effectively prevents bleeding in patients with AHA and that the initiation of immunosuppressive therapy can be delayed while receiving this treatment.

• #1 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.

• #1 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

  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. […] 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.

• #1 AHCDO prophylaxis study – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-200-december-2017/ahcdo-prophylaxis-study/

  In the June issue of National Haemophilia, I provided an outline of the large retrospective study conducted last year characterising current prophylaxis practice in patients with haemophilia A and haemophilia B in Australia. […] Overall 453 patients (82%) with severe haemophilia A were receiving regular prophylaxis, versus 42 patients (25%) with moderate haemophilia A. There were 66 patients (75%) with severe haemophilia B versus 11 patients (13%) with moderate haemophilia B receiving regular prophylaxis. […] Near universal prophylaxis was achieved in the paediatric group. The percentage of patients on prophylaxis was generally seen to decline with increasing age. […] Overall, rates of patients with severe haemophilia on prophylaxis in Australia are generally comparable with other developed nations.

• #1 Hemophilia Review

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

  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. Factor concentrates with a short half-life lead to more frequent IV administration, reduced patient adherence, and increased treatment cost. […] Another significant obstacle that hemophilia patients face is the development of inhibitors. Inhibitors, which are immunoglobulin G antibodies that specifically target factor VIII, can be completely inactivating or incompletely inactivating. Completely inactivating inhibitors commonly develop in patients with severe hemophilia A and lead to more frequent and serious bleeding complications. Hemlibra, a factor IXa and factor X-directed antibody agent, is FDA approved for prophylaxis of hemophilia A patients who have inhibitors.

• #1 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

  Despite the advantages of prophylactic treatment for hemophilia, patients tend to discontinue or not adhere to it because of several challenges such as long-term use, high cost, young patients transitioning to adolescents, and switch to self-infusion or self-care. […] The majority of studies reported lower annualized bleeding rates in patients receiving prophylaxis compared with those receiving on-demand treatment or those who discontinued prophylaxis. […] In developing countries like China, data suggest that low doses were administered because of limited available resources. However, standard dose or individualized prophylaxis should be provided to prevent joint damage in the long term. […] This systematic review emphasizes the importance of adherence to prophylaxis among young adults transitioning from childhood.

• #1 Adherence to Prophylaxis in Adolescents and Young Adults with Severe Haemophilia: A Quantitative Study with Patients | PLOS One

  https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0169880

  The findings suggest that interventions aimed at improving adherence should particularly consider how they may improve social support for patients, increase patients necessity/concern differential scores and positive outcome expectations. It is also important to consider that negative emotions, such as fear, may for some patients work as a motivator to keep on track with prophylaxis.

• #1 Medication Adherence for Haemophilia Patients: Outcome of Prophylaxis Treatment Intervention

  https://www.mdpi.com/2227-9032/9/12/1702

  The prophylactic factor regimens have demonstrated clinical advantages in preventing and reducing bleeding episodes. […] This study also aims to understand the relationship between medication adherence, bleeding, and comorbidity. […] The pharmacist-run HMTAC has proven to yield a high level of adherence post a minimum of three months of enrolment. […] Through the HMTAC service, pharmacists play a crucial role in imparting medication compliance and knowledge, recommending therapy regimens, and intervening in medication-related problems. […] The adherence level showed a significant association with ABR in the VERITAS-Pro total score. […] This study also highlights the significant correlation between post-HMTAC run by pharmacist adherence levels with ABR and comorbidities.

• #1 Exploring haemophilia prophylaxis – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-226-june-2024/exploring-haemophilia-prophylaxis/

  One of the most memorable sessions at WFH 2024 World Congress focused on the optimal dosage of prophylaxis treatment for individuals with haemophilia, highlighting the importance of this treatment in preventing bleeding episodes and joint damage. […] In the session, the speakers mentioned that prophylaxis treatment dosages for individuals with haemophilia vary across different countries, with more economically developed countries often offering higher doses and more frequent treatments due to better access to resources and healthcare infrastructure. […] The initial haemophilia treatment goal was to respond to acute bleeds, often with episodic treatment. Then prophylaxis with a target of a factor level greater than 1% was introduced to reduce bleeding for patients with a severe phenotype, and later an increased target of a factor level above 3-5% was introduced to prevent bleeds and preserve joint health.

• #1 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 can still be individualized to affordability. […] Higher protective trough level can be achieved by using smaller doses given more frequently without an increase in consumption/cost.

• #1 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

  Central to the success of prophylaxis are clinics with comprehensive care that provide the necessary professional expertise, support, and counseling, to educate patients, families, and other healthcare professionals, and to support research for improved hemophilia care. […] 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. […] Individualized prophylaxis by understanding bleeding triggers and PK profiles would allow us to target appropriate trough levels that can achieve zero bleeds, provide the greatest chance of preserving joint health, and ultimately meet the specific goals for each patient. […] 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. […] A dedicated hemophilia clinic with comprehensive care remains fundamental to prophylaxis irrespective of economic capacity, and should also be the priority in all developing nations.

• #1 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).

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

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

  Since it is an inherited condition, the best way to prevent this condition is to ensure that those with history of hemophilia go through pre-conception screening. […] 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. 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.

• #1 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.

• #1 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.

• #1 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.

• #1 Prophylaxis – Haemophilia Wales

  https://haemophiliawales.org/prophylaxis/

  Prophylaxis is recommended for all children with severe factor deficiencies. Prophylaxis involves small regular infusions of factor concentrate to prevent spontaneous bleeding and to minimise traumatic bleeding. This treatment regime although difficult to start with will prevent joint damage and will lead to improved quality of life. […] Both preventative and on demand treatment can be administered at home. The benefits of self infusion at home means that bleeds can be treated quickly and this reduces disruption to normal family life. […] Education from a young age is always an important factor in bringing up children with Haemophilia.

• #1 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 WFH recommends initiation of prophylaxis at any age to reduce hemarthrosis and slow down the progression of hemophilic arthropathy. […] As per National Hemophilia Foundation (NHF) guidelines, patients with hemophilia benefit from lifelong preventive treatment. […] The WFH recommends age-specific hemophilia camps to understand the importance of adherence to prophylaxis and develop self-infusion skills. […] 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 results indicate that non-adherence to prophylaxis among young adults transitioning from childhood can lead to high ABRs and joint deterioration. Hence, adherence to prophylaxis is critical among adults. Primary prophylaxis gives better clinical outcomes while secondary and tertiary prophylaxis is better than on-demand treatment. In countries like China, low-dose prophylaxis can help in preventing joint bleeds in the short term compared with on-demand treatment. However, in the long term and with improvement in economic conditions, standard-dose therapy or individualized prophylaxis is suggested as it preserves joint health and improves the quality of life, in turn helping in high adherence to treatment among young adults.

• #1 Prevention of Hemophilia: Is It Possible?

  https://www.healthline.com/health/prevention-of-hemophilia

  Hemophilia is a genetic condition that cant be prevented. However, modern medical treatments can help prevent bleeding and increase life span. […] However, modern treatments can prevent excess bleeding and improve survival for people with hemophilia. […] Preventing bleeding is typically a part of hemophilia treatment. This is called prophylaxis: It prevents both bleeding from minor injuries and damage to joints that can happen when hemophilia causes internal bleeding. […] Often, prevention treatments are infusions of medication that help blood clot. […] However, clotting treatments can help people with hemophilia prevent bleeding. These treatments are helping people with hemophilia live longer than ever before.

• #1 Hemophilia Basics

  https://www.webmd.com/a-to-z-guides/understanding-hemophilia-basics

  Because its a genetic disease, theres nothing you can do to prevent hemophilia. But you can get genetic testing to see if youre a carrier before you decide to have a baby. Some parents who have the gene choose in-vitro fertilization so that an embryo can be tested for the disease before its implanted.

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

  https://www.patientsengage.com/conditions/hemophilia/prevention?page=1

  Since it is an inherited condition, the best way to prevent this condition is to ensure that those with history of hemophilia go through pre-conception screening. […] 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. […] Regular medical checkups and tests for monitoring health status and follow-up of prophylactic treatment are very important to avoid complications. […] Use protective devices to avoid injuries such as helmets during physical activities or driving to minimize the risk of traumatic injuries. […] 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.

• #1 What Is Hemophilia?

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

  Healthcare providers treat hemophilia by replacing missing clotting factors. […] Providers are researching gene therapy and gene replacement therapy as new ways to treat and possibly cure hemophilia. […] 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.

• #1 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).

• #2 Adherence to Prophylaxis in Adolescents and Young Adults with Severe Haemophilia: A Quantitative Study with Patients | PLOS One

  https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0169880

  haemophilia is an inherited bleeding disorder caused by a deficiency in one of the blood coagulation factors. For people affected by severe haemophilia, the deficiency can cause spontaneous internal bleeding. Most young people with severe haemophilia in the UK follow a preventative treatment regimen (prophylaxis) consisting of several intravenous injections of factor concentrate each week. There is good evidence that prophylaxis reduces bleeds whilst also improving quality of life. […] United Kingdom Haemophilia Centre Doctors Organization (UKHCDO) guidelines recommend early implementation of prophylaxis for children with severe haemophilia, and that adolescents and adults should be encouraged to continue regular prophylaxis at least until they have reached physical maturity. Adherence to a prophylactic regimen is essential to ensure that the full benefits (i.e. prevention of bleeding) are realised.

• #2 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. […] Prophylaxis may be initiated with a dose-escalation strategy, starting at once weekly dosing, and then increasing as needed based on bleed frequency.

• #2 Prophylaxis is the new standard of care in patients with haemophilia

  http://www.scielo.org.za/scielo.php?script=sci_arttext&pid=S0256-95742022000600007

  The therapeutic goal of prophylaxis is to prevent spontaneous bleeding with consequent preservation of joint health. […] The long-term benefits of prophylaxis include less arthropathy, less disability, and improved quality of life. […] The new non-factor therapies, such as emicizumab, have enabled the adoption of prophylaxis as the new standard of care in haemophilia.

• #2 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

  The concept of prophylaxis aims to convert severe hemophilia to moderate/mild hemophilia by the infusion of clotting factor on a regular basis (often 2 to 3 times per week), even in the absence of a bleed. […] The World Federation of Hemophilia (WFH) and the Medical and Scientific Advisory Commission of the National Hemophilia Foundation recommend the use of prophylaxis to prevent bleeding and joint destruction and preserve musculoskeletal function. […] Prophylaxis was found to be more effective when started early, confirming the effectiveness in preventing bleeds and joint arthropathy. […] It is recommended that prophylaxis be initiated early and factor levels maintained at more than 1% of normal at all times. […] Studies have shown that the earlier prophylaxis is initiated, the greater the likelihood of preventing joint damage.

• #2 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.

• #2 Exploring haemophilia prophylaxis – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-226-june-2024/exploring-haemophilia-prophylaxis/

  I see this evolution of haemophilia treatment as being corelated to prevention as the core of health care. […] Dr Maria Elisa Mancuso, a haematologist from Italy, suggested based on her research findings that prophylaxis treatment should maintain a high factor VIII (8) trough level at 40% or above to achieve zero bleeds and that prophylaxis treatment should also be available for mild haemophilia patients. […] Dr Carcao suggested that prophylaxis treatment should be started as early as possible to achieve joint health preservation and minimise bleeds. […] However, he noted that prophylaxis treatment in haemophilia is expensive and around 80% of the world has very little or zero access to the products. […] Consequently, although low dose prophylaxis tailored to individual needs and affordability are not enough, they are much better than no prophylaxis.

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

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

  Prophylaxis has been shown in many studies to prevent or at least reduce the progression of damage to target sites, such as joints. […] According to a review of six randomized, controlled trials, preventive therapy started early in childhood, as compared with on-demand treatment, can reduce total bleeds and bleeding into joints, thus decreasing overall joint deterioration and improving patients’ quality of life. […] Indeed, the International Society on Thrombosis and Haemostasis (ISTH) Hemophilia Guideline Panel recommends that patients with severe and moderately severe congenital hemophilia B without inhibitors be treated prophylactically rather than through episodic management of bleeding events. […] 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).

• #2 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

  Continuous prophylaxis is defined as treatment that is initiated with the intent of treating for 52 weeks of the year and is accomplished for at least 45 weeks of that year. […] Secondary prophylaxis is a treatment regimen initiated after 2 or more bleeds into large joints but before the onset of joint disease documented by physical examination and imaging studies. […] Studies in adolescents and adults with hemophilia have shown significant benefits of secondary prophylaxis. […] Prophylaxis was also associated with significantly fewer target joints and better health-related quality of life (HRQoL). […] 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.

• #2 Prophylaxis | The Haemophilia Society

  https://haemophilia.org.uk/bleeding-disorders/treatment-types/prophylaxis/

  Prophylaxis is the name given to regular treatment to prevent bleeds. For someone with haemophilia, this is treatment, typically 3 times a week, which helps the blood clot and minimises the likelihood of bleeds and long-term joint damage. […] Prophylaxis is hugely important for people living with haemophilia. Severe haemophiliacs who experience spontaneous bleeds into joints may sustain long-term damage such as arthritis, with certain joints becoming more vulnerable to target joints.

• #2

  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. […] In patients with severe hemophilia A, prophylaxis with replacement FVIII can prevent bleeds and structural joint damage when initiated at a young age, and can decrease bleeding frequency, slow joint disease progression, and improve quality of life – even when initiated in adults with established joint damage. […] 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.

• #2 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.

• #2 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).

• #2 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. […] Hympavzi provided similar bleeding rates.

• #2 Exploring haemophilia prophylaxis – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-226-june-2024/exploring-haemophilia-prophylaxis/

  Non-factor treatment is an option for personalised prophylaxis treatment, but raises questions such as the risk of thrombosis, cost and how individuals will handle the variety of medications when experiencing a bleed. […] Dr Veena Selvaratnam, a haematologist from Malaysia, presented the results on her low dose emicizumab studies, showing that low dose emicizumab resulted in zero bleeds in 9 out of 10 patients over 37- 293 weeks, and their QoL (quality of life) score improved significantly.

• #2 AHCDO prophylaxis study – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-200-december-2017/ahcdo-prophylaxis-study/

  In the June issue of National Haemophilia, I provided an outline of the large retrospective study conducted last year characterising current prophylaxis practice in patients with haemophilia A and haemophilia B in Australia. […] Overall 453 patients (82%) with severe haemophilia A were receiving regular prophylaxis, versus 42 patients (25%) with moderate haemophilia A. There were 66 patients (75%) with severe haemophilia B versus 11 patients (13%) with moderate haemophilia B receiving regular prophylaxis. […] Near universal prophylaxis was achieved in the paediatric group. The percentage of patients on prophylaxis was generally seen to decline with increasing age. […] Overall, rates of patients with severe haemophilia on prophylaxis in Australia are generally comparable with other developed nations.

• #2 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.

• #2 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.

• #3 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

  Continuous prophylaxis is defined as treatment that is initiated with the intent of treating for 52 weeks of the year and is accomplished for at least 45 weeks of that year. […] Secondary prophylaxis is a treatment regimen initiated after 2 or more bleeds into large joints but before the onset of joint disease documented by physical examination and imaging studies. […] Studies in adolescents and adults with hemophilia have shown significant benefits of secondary prophylaxis. […] Prophylaxis was also associated with significantly fewer target joints and better health-related quality of life (HRQoL). […] 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.

• #3 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.

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