Materiały źródłowe

• #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. […] Replacement clotting factor can be made from donated blood. Similar products, called recombinant clotting factors, are made in a laboratory, not from human blood. […] Other therapies include: Desmopressin. In some forms of mild hemophilia, this hormone can stimulate the body to release more clotting factor. It can be injected slowly into a vein or used as a nasal spray. […] Emicizumab (Hemlibra). This is a newer drug that doesn’t include clotting factors. This drug can help prevent bleeding episodes in people with hemophilia A.

• #2 Treatment of Hemophilia | Hemophilia | CDC

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

  If you have hemophilia, you need treatment so your blood can clot. […] Treatment products have different ways of helping blood clot. […] Hemophilia treatment centers specialize in caring for people with bleeding disorders. […] 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. […] For hemophilia A, another treatment method is a non-factor product that stands in for, or replaces the function of, the missing factor VIII. This treatment is given by injection under the skin that can be given by people with hemophilia themselves. […] Healthcare providers typically prescribe treatment products for episodic care or prophylactic care. Episodic care is used to stop a patients bleeding episodes; 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. Giving factor treatment products at home means that bleeds can be treated more quickly, resulting in less serious bleeding and fewer side effects.

• #3 How Is Hemophilia Treated? | Hematology-Oncology Associates of CNY

  https://www.hoacny.com/patient-resources/blood-disorders/what-hemophilia/how-hemophilia-treated

  The main treatment for hemophilia is called replacement therapy. Concentrates of clotting factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are slowly dripped or injected into a vein. These infusions help replace the clotting factor that’s missing or low. […] You may have replacement therapy on a regular basis to prevent bleeding. This is called preventive or prophylactic (PRO-fih-lac-tik) therapy. Or, you may only need replacement therapy to stop bleeding when it occurs. This use of the treatment, on an as-needed basis, is called demand therapy. […] Demand therapy is less intensive and expensive than preventive therapy. However, there’s a risk that bleeding will cause damage before you receive the demand therapy. […] Complications of replacement therapy include: Developing antibodies (proteins) that attack the clotting factor, Developing viral infections from human clotting factors, Damage to joints, muscles, or other parts of the body resulting from delays in treatment.

• #4 Treatment of Hemophilia | Hemophilia | CDC

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

  Good quality medical care from healthcare providers who know a lot about the disorder can help people with hemophilia prevent some serious problems. This type of specialty care is at a comprehensive hemophilia treatment center (HTC). An HTC provides patients with the care and education to address all issues related to the disorder. […] The two main types of clotting factor concentrates available are plasma-derived factor concentrates and recombinant factor concentrates. […] Hemlibra works by replacing the function of factor VIII, rather than replacing the missing clotting factor VIII directly. It can be used to either prevent or reduce the frequency of bleeding episodes in people with hemophilia A. […] DDAVP and Stimate are medications that are similar to a hormone that occurs naturally in the body. The medications release factor VIII from where it is stored in the body tissues.

• #5 How Is Hemophilia Treated? | Hematology-Oncology Associates of CNY

  https://www.hoacny.com/patient-resources/blood-disorders/what-hemophilia/how-hemophilia-treated

  The main treatment for hemophilia is called replacement therapy. Concentrates of clotting factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are slowly dripped or injected into a vein. These infusions help replace the clotting factor that’s missing or low. […] You may have replacement therapy on a regular basis to prevent bleeding. This is called preventive or prophylactic (PRO-fih-lac-tik) therapy. Or, you may only need replacement therapy to stop bleeding when it occurs. This use of the treatment, on an as-needed basis, is called demand therapy. […] Demand therapy is less intensive and expensive than preventive therapy. However, there’s a risk that bleeding will cause damage before you receive the demand therapy. […] Complications of replacement therapy include: Developing antibodies (proteins) that attack the clotting factor, Developing viral infections from human clotting factors, Damage to joints, muscles, or other parts of the body resulting from delays in treatment.

• #6 Haemophilia

  https://www.nhs.uk/conditions/haemophilia/

  The clotting factor used to treat haemophilia today is very safe. Much of it’s synthetic and has no risk of infection. When it’s made from donated blood, it’s tested and treated to make sure it’s as safe as possible, and the risk of getting an infection from it is very low. […] Your haemophilia team will usually show you how to treat bleeding yourself at home, with clotting factor injections or other medicines. […] Treatment reduces the risk of complications.

• #7 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. […] Replacement clotting factor can be made from donated blood. Similar products, called recombinant clotting factors, are made in a laboratory, not from human blood. […] Other therapies include: Desmopressin. In some forms of mild hemophilia, this hormone can stimulate the body to release more clotting factor. It can be injected slowly into a vein or used as a nasal spray. […] Emicizumab (Hemlibra). This is a newer drug that doesn’t include clotting factors. This drug can help prevent bleeding episodes in people with hemophilia A.

• #8 Haemophilia Treatment | Medica Superspecialty Hospital

  https://medicahospitals.in/blog/haemophilia-symptoms-diagnosis-and-treatment/

  Haemophilia is a bleeding disorder in which the blood does not clot properly. […] The ideal way to treat haemophilia is to replace the missing blood clotting factor, so that the blood can clot properly. This is done by injecting treatment products, known as clotting factor concentrates, into an individuals vein. […] Clinicians typically prescribe treatment products for episodic care or prophylactic care. Episodic care is used to stop an individuals bleeding episodes; prophylactic care is used to prevent bleeding episodes from occurring. […] Today, its possible for people with haemophilia, and their families, to learn how to give their own clotting factor treatment products at home. Giving factor treatment products at home indicates that bleeds can be treated faster, resulting in comparatively less serious bleeding and fewer side effects.

• #9 Treating Hemophilia B | Bleeding Disorders

  https://www.bleedingdisorders.com/hemophilia-b/treatment

  Fewer visits to the healthcare provider (HCP) or emergency room are needed. Home treatment costs less than treatment in a medical care setting. […] Home treatment helps children accept treatment and take responsibility for their own health. […] Today, clotting factor IX concentrates can be made without human plasma. Infusions are called recombinant clotting factors. […] In the past 25+ years, significant advances have been made in the manufacturing of these recombinant clotting factors. […] Recombinant technology has helped produce factor concentrates that avoid the risk of human-viral contamination. […] The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) encourages the use of recombinant clotting factor products. Your HCP or your hemophilia treatment center (HTC) will help you decide which is right for you. […] Factor treatment has to be personalized to your lifestyle.

• #10 Haemophilia Treatment – Irish Haemophilia Society

  https://haemophilia.ie/about-bleeding-disorders/haemophilia/haemophilia-treatment-2/

  Haemophilia treatment in Ireland has improved hugely in the last decade. There are excellent preventative treatment options for individuals with haemophilia. […] Unlike 70% of people with bleeding disorders across the world, patients in Ireland have access to the safest and most effective haemophilia treatment available. The treatment of bleeding disorders has improved over the last decade with new types of products changing the way we think about treatment. There are two basic types of treatment approaches: […] On demand– This is treatment received in response to a bleed which may have occurred after an injury or accident or may have been spontaneous. The aim of this is to stop the bleed once it has started. Each bleeding episode must be promptly treated by an intravenous (into a vein) infusion of the clotting factor which is deficient. Once the bleeding stops, pain rapidly diminishes and use of the limb returns. The requirement for rapid treatment has meant that the vast majority of treatment in those with severe haemophilia are treated at home where the person with haemophilia or the parent has been trained to administer the treatment. In addition, planned on-demand treatment may also be used prior to some dental treatment, surgery, or childbirth.

• #11 Haemophilia Treatment – Irish Haemophilia Society

  https://haemophilia.ie/about-bleeding-disorders/haemophilia/haemophilia-treatment-2/

  Prophylaxis– This is treatment taken regularly, to prevent bleeding from an injury before it requires further interventions. This is where the most advances have been made in the bleeding disorder community to make it easier to protect from bleeding and reduce the impact of even 1 or 2 bleeds a year, on long term joint movement and pain. […] Standard half-life treatment is a prophylactic treatment option. With SHL, the missing clotting factor concentrates are injected into the blood and stay there for a relatively short period of time, therefore requiring repeat injections – every 2-3 days for Haemophilia A and twice a week for Haemophilia B. […] These products stay in the bloodstream for longer than SHL products. Therefore people may choose to infuse less often than with SHL, meaning that those with Haemophilia A would need to infuse twice per week while those with Haemophilia B would need to infuse once per week, once every ten days or once every two weeks.

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

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

  Patients can be treated with prophylaxis or with intermittent, on-demand therapy for bleeding events. 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, resulting in decreased overall joint deterioration and improved 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 A without inhibitors be treated prophylactically rather than through episodic management of bleeding events.

• #13 Haemophilia

  https://www.nhs.uk/conditions/haemophilia/

  Haemophilia is usually treated with injections of clotting factor to replace the missing clotting factor in your blood. […] It can also be treated with other medicines including emicizumab, desmopressin and tranexamic acid. […] Haemophilia is treated in 2 main ways: […] If you have severe haemophilia, you’ll usually need injections up to 3 times a week to prevent bleeding (preventative or prophylactic treatment). You can usually do these injections yourself at home. […] If you have mild or moderate haemophilia, you’ll usually only need treatment to stop or reduce bleeding after it starts (on-demand treatment). You may also need treatment before having surgery or dental work. […] Some people develop antibodies to clotting factor, called inhibitors, which make this treatment less effective. If this happens, your haemophilia team will work with you to find you a more suitable treatment.

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

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

  Patients may be treated with prophylaxis or with intermittent, on-demand therapy for bleeding events. 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.

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

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

  Patients can be treated with prophylaxis or with intermittent, on-demand therapy for bleeding events. 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, resulting in decreased overall joint deterioration and improved 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 A without inhibitors be treated prophylactically rather than through episodic management of bleeding events.

• #16 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. […] The treatment of patients with inhibitors of FVIII is difficult. Bleeding episodes in patients with low-titer inhibitors (ie, concentrations below 5 Bethesda units [BU]) occasionally can be overcome with high doses of factor VIII.

• #17 Hemophilia therapy: the future has begun

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7049365/

  Alongside this, the last decade has witnessed further spectacular therapeutic progress, such as the availability of coagulation factors with a longer plasma half-life that allow for wider intervals between treatment. […] Moreover, new therapeutic products based on new mechanisms other than the replacement of the deficient factor, have become available (emicizumab) or are at an advanced stage of development. […] This review celebrates the success story of hemophilia care, while also discussing current limitations, issues and as yet unmet needs. […] The prospects of cure by means of gene therapy are also outlined. […] Primary prophylaxis of bleeding episodes became the evidence-based standard of care following the randomized clinical trial of Manco-Johnson et al., who demonstrated that this preventive regimen was clearly superior to the episodic management of bleeds, because it reduced the rate of their occurrence and also achieved a marked reduction in joint damage.

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

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

  Patients can be treated with prophylaxis or with intermittent, on-demand therapy for bleeding events. 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, resulting in decreased overall joint deterioration and improved 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 A without inhibitors be treated prophylactically rather than through episodic management of bleeding events.

• #19 Haemophilia Treatment – Current and Future Challenges – touchONCOLOGY

  https://touchoncology.com/haematology/journal-articles/haemophilia-treatment-current-and-future-challenges/

  The importance of immediate prophylaxis after the initial bleeding episode is well documented: the longer the delay in prophylactic treatment following the first bleed, the higher the risk of developing arthropathy. […] Studies have shown that this approach uses less factor VIII (FVIII) compared with traditional prophylaxis. […] The obvious benefit in adopting a prophylactic regimen is the reduction of joint bleeds. […] Prophylaxis in children with haemophilia therefore presents a beneficial outcome in terms of preventing joint damage, reducing the risk of inhibitor development and, as a result, improving overall quality of life, allowing children to be children and to live the lives they choose. […] Prophylaxis in adults with severe or moderate haemophilia is growing in popularity.

• #20 Hemophilia therapy: the future has begun

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7049365/

  A subsequent randomized study by Gringeri et al. confirmed and strengthened this evidence, so that prophylaxis became the undisputed standard of care in countries that could afford it. […] Additional and important advantages were a much improved patient quality of life, including less hospitalizations and days lost from school and work, and an improved social life. […] However, the implementation of prophylaxis met some obstacles, in addition to that of affordability. […] The degree of adherence was often less than optimal, particularly in children and adolescents, owing to the burden created by the need of 2-3 or more weekly intravenous injections. […] Two extended half-life (EHL) recombinant coagulation factors were licensed in 2014: the Fc-fused FIX eftrenonacog alfa and the Fc-fused FVIII efmoroctocog alfa.

• #21 Haemophilia treatment today

  https://www.haemevolution.eu/patient/treatment-developments

  People with haemophilia now have access to newer medications, like extended half-life (EHL) products, which decrease the frequency of injections. […] Many people with haemophilia require regular intravenous infusions of factor replacement. This is because people with moderate to severe haemophilia have less than 1% to 5% of factor levels compared with 40% to 150% for people without haemophilia. To maintain near normal factor levels, people with haemophilia require infusions with replacement factors. Depending on factor levels, some require intravenous infusions as often as every other day. […] Therapies for haemophilia have been continuously improving, becoming more effective at controlling symptoms while reducing the frequency of treatment. For example, extended half-life (EHL) products have been introduced, which require fewer infusions.

• #22 Hemophilia therapy: the future has begun

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7049365/

  EHL FIX products are much more satisfactory, because they can be given every 10 or even 15 days, and thus allow a lower annual burden of intravenous injections, the average reduction being more prominent (60%) than for FVIII products (~30%). […] Furthermore, higher trough levels of both FVIII (2-3%) and FIX (5-10%) could be achieved than with SHL products. […] In spite of the progress made with the availability of EHL factors, unmet needs remained. […] In HA patients without inhibitors, the reduction in the frequency of intravenous injections was not considered satisfactory, and therapy still based on the need for a venous access continued to be unattractive. […] With these drawbacks in mind, therapeutic approaches that were not based on the replacement of the deficient factor were developed.

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

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

  Prophylaxis is not universally accepted, with only about half the children with hemophilia A receiving this treatment modality in the United States. […] 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. […] 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.

• #24 Treatment of hemophilia | Hemophilia

  https://www.hemophilia.ca/treatment-of-hemophilia/

  Unfortunately, the replacement of the missing clotting factor is not permanent. With standard half-life factor, the rise in clotting factor activity following infusion gradually diminishes and is half gone after 12 to 24 hours. This means that within 2 or 3 days, almost none of the medication remains in the blood and the person with hemophilia will have blood clotting problems again unless they receive another infusion of clotting factor. Extended half-life factor concentrates, introduced in Canada in 2016, last longer in the bloodstream. The median extended half-life of factor VIII is 16 to 18 hours, and 50 to 100 hours for factor IX. […] Non-factor replacement therapy is a type of treatment that is not based on the use of factor concentrates to replace factor VIII missing in people with hemophilia A and factor IX missing in people with hemophilia B. Non-factor therapies work differently – they are recombinant blood proteins or molecules engineered to perform the action of missing clotting factor or rebalance and restore coagulation capacity to people with hemophilia.

• #25 Advances in the management of haemophilia: emerging treatments and their mechanisms | Journal of Biomedical Science | Full Text

  https://jbiomedsci.biomedcentral.com/articles/10.1186/s12929-021-00760-4

  Emicizumab, a recombinant humanised bispecific IgG antibody, mimics the cofactor function of the missing FVIII in HA. It simultaneously binds activated FIX (FIXa) and factor X (FX), bringing them into spatial proximity to promote FIXa-catalysed FX activation, thereby restoring haemostasis. […] The unique molecular structure of emicizumab means it is not subject to the same physiologic mechanisms that eliminate FVIII from circulation, hence it persists in circulation for much longer compared to FVIII. It has a long half-life of around 45 weeks, which supports dosing regimens once weekly, every 2 weeks or even monthly. […] Fitusiran (ALN-AT3), a novel therapy applicable to both HA and HB, consists of the amino acid, N-Acetylgalactosamine (GalNAc), the ligand of the hepatic asialoglycoprotein receptors, conjugated to a synthetic siRNA. Administered subcutaneously, it targets and degrades a region of the SERPINC1 gene mRNA, preventing antithrombin production and enhancing thrombin generation.

• #26 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. […] Replacement clotting factor can be made from donated blood. Similar products, called recombinant clotting factors, are made in a laboratory, not from human blood. […] Other therapies include: Desmopressin. In some forms of mild hemophilia, this hormone can stimulate the body to release more clotting factor. It can be injected slowly into a vein or used as a nasal spray. […] Emicizumab (Hemlibra). This is a newer drug that doesn’t include clotting factors. This drug can help prevent bleeding episodes in people with hemophilia A.

• #27 Treatment of Hemophilia | Hemophilia | CDC

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

  Good quality medical care from healthcare providers who know a lot about the disorder can help people with hemophilia prevent some serious problems. This type of specialty care is at a comprehensive hemophilia treatment center (HTC). An HTC provides patients with the care and education to address all issues related to the disorder. […] The two main types of clotting factor concentrates available are plasma-derived factor concentrates and recombinant factor concentrates. […] Hemlibra works by replacing the function of factor VIII, rather than replacing the missing clotting factor VIII directly. It can be used to either prevent or reduce the frequency of bleeding episodes in people with hemophilia A. […] DDAVP and Stimate are medications that are similar to a hormone that occurs naturally in the body. The medications release factor VIII from where it is stored in the body tissues.

• #28 Advances in the management of haemophilia: emerging treatments and their mechanisms | Journal of Biomedical Science | Full Text

  https://jbiomedsci.biomedcentral.com/articles/10.1186/s12929-021-00760-4

  Emicizumab, a recombinant humanised bispecific IgG antibody, mimics the cofactor function of the missing FVIII in HA. It simultaneously binds activated FIX (FIXa) and factor X (FX), bringing them into spatial proximity to promote FIXa-catalysed FX activation, thereby restoring haemostasis. […] The unique molecular structure of emicizumab means it is not subject to the same physiologic mechanisms that eliminate FVIII from circulation, hence it persists in circulation for much longer compared to FVIII. It has a long half-life of around 45 weeks, which supports dosing regimens once weekly, every 2 weeks or even monthly. […] Fitusiran (ALN-AT3), a novel therapy applicable to both HA and HB, consists of the amino acid, N-Acetylgalactosamine (GalNAc), the ligand of the hepatic asialoglycoprotein receptors, conjugated to a synthetic siRNA. Administered subcutaneously, it targets and degrades a region of the SERPINC1 gene mRNA, preventing antithrombin production and enhancing thrombin generation.

• #29 Haemophilia Treatments

  https://www.rch.org.au/clinicalguide/guideline_index/Haemophilia_treatments/

  RCH Health Professionals Clinical Practice Guidelines Haemophilia Treatments […] Clotting factor replacement […] Some haemophilia treatment products are stored in hospital pharmacies. Others are stored in hospital or state blood banks […] Consider the availability of a product before prescribing. More information here […] Emicizumab (Hemlibra) is a bi-specific monoclonal antibody directed toward factor IXa and factor X, and acts to mimic the function of missing factor VIII […] Effective in children with haemophilia A, with or without factor VIII inhibitor […] Administered via subcutaneous injection […] Not intended to treat acute bleeding episodes […] Antifibrinolytic treatment reduces breakdown of blood clots and is effective for treating and preventing the recurrence of mouth bleeds and epistaxis in all severities of haemophilia

• #30 New, lifechanging hemophilia treatments improve patients’ health, outlook | Discovery | UT Southwestern Medical Center

  https://utswmed.org/medblog/hemophilia-adult-treatment/

  New hemophilia treatments and targeted physical therapy make it easier for patients to live more active lives. […] Today, our patients at the North Texas Comprehensive Hemophilia Treatment Center the only adult-focused hemophilia treatment center in Texas have access to treatments that patients are describing as life changing. […] But after more than three decades of limited treatment options, several new and improved therapies have become available in the last few years, such as single-injection therapies with no IV required. And with advanced therapies coming down the pike, the future is bright for patients living with hemophilia. […] In 2017, the U.S. Food and Drug Administration (FDA) approved a new single-injection drug called emicizumab (brand name Hemlibra), to prevent or reduce the frequency of bleeding episodes.

• #31 New, lifechanging hemophilia treatments improve patients’ health, outlook | Discovery | UT Southwestern Medical Center

  https://utswmed.org/medblog/hemophilia-adult-treatment/

  Instead of having to find a vein for multiple IV infusions a week, emicizumab is given through an injection under the skin once a week, once every two weeks, or once a month. […] This has made a huge difference in terms of the burden of the treatment on our patients. […] However, we expect a new once-a-month subcutaneous hemophilia injection, Fitusiran, to be available soon for patients with hemophilia A and those who are missing clotting factor IX (hemophilia B). […] While several types of gene therapies are being studied in clinical trials, gene transfer via adeno-associated virus (AAV) vector-based gene therapy is furthest along the pipeline. […] AAV vector-based gene therapy uses a modified virus to deliver functional copies of factor VIII or factor IX genes to cells in the liver, empowering the body to make its own clotting factor.

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

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

  Prophylaxis is not universally accepted, with only about half the children with hemophilia A receiving this treatment modality in the United States. […] 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. […] 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.

• #33 Treatment of hemophilia | Hemophilia

  https://www.hemophilia.ca/treatment-of-hemophilia/

  Hemlibra is a bispecific monoclonal antibody – a type of protein made in the laboratory that can bind to factor IXa and factor X in the blood. It is not a factor protein but it mimics and performs the same function as factor VIII in the clotting process. It is also called a factor VIII mimetic. […] Hemlibra offers several advantages compared to factor concentrates. […] Gene therapy for hemophilia is a promising new option first approved in 2023. It is a one-time treatment that enables a person to produce factor VIII or IX on their own, but it is not a full cure and not for everyone. […] The factor VIII mimetic Hemlibra provides very effective prophylaxis for hemophilia A. It provides a steady clotting factor activity level equivalent to about 15% factor VIII and results in over 90% fewer bleeds in most people with severe hemophilia A.

• #34 Haemophilia Treatments

  https://www.rch.org.au/clinicalguide/guideline_index/Haemophilia_treatments/

  RCH Health Professionals Clinical Practice Guidelines Haemophilia Treatments […] Clotting factor replacement […] Some haemophilia treatment products are stored in hospital pharmacies. Others are stored in hospital or state blood banks […] Consider the availability of a product before prescribing. More information here […] Emicizumab (Hemlibra) is a bi-specific monoclonal antibody directed toward factor IXa and factor X, and acts to mimic the function of missing factor VIII […] Effective in children with haemophilia A, with or without factor VIII inhibitor […] Administered via subcutaneous injection […] Not intended to treat acute bleeding episodes […] Antifibrinolytic treatment reduces breakdown of blood clots and is effective for treating and preventing the recurrence of mouth bleeds and epistaxis in all severities of haemophilia

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

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

  Prophylaxis is not universally accepted, with only about half the children with hemophilia A receiving this treatment modality in the United States. […] 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. […] 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.

• #36 Haemophilia | Better Health Channel

  https://www.betterhealth.vic.gov.au/health/conditionsandtreatments/haemophilia

  Current treatment for people with moderate or severe haemophilia aims to prevent bleeding, pain and joint damage. […] In Australia most children and young people with severe haemophilia are treated with emicizumab. […] People with mild haemophilia will not need prophylaxis and will only need treatment for a bleed when it occurs, usually after trauma or injury, or when they are preparing for surgery or some dental work.

• #37 Treatment of Hemophilia | Hemophilia | CDC

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

  Good quality medical care from healthcare providers who know a lot about the disorder can help people with hemophilia prevent some serious problems. This type of specialty care is at a comprehensive hemophilia treatment center (HTC). An HTC provides patients with the care and education to address all issues related to the disorder. […] The two main types of clotting factor concentrates available are plasma-derived factor concentrates and recombinant factor concentrates. […] Hemlibra works by replacing the function of factor VIII, rather than replacing the missing clotting factor VIII directly. It can be used to either prevent or reduce the frequency of bleeding episodes in people with hemophilia A. […] DDAVP and Stimate are medications that are similar to a hormone that occurs naturally in the body. The medications release factor VIII from where it is stored in the body tissues.

• #38 Haemophilia Treatments

  https://www.rch.org.au/clinicalguide/guideline_index/Haemophilia_treatments/

  Management of children with inhibitors is complex and the haemophilia treatment centre should be consulted […] Treatment and prevention of bleeding in children with inhibitors is managed with bypassing agents eg NovosevenRT, FEIBA […] DDAVP releases stored factor VIII and von Willebrand factor into the circulation […] It is used in children with mild haemophilia A where there is a documented record of safe and satisfactory response to a Desmopressin challenge […] It is not adequate as a single agent to achieve haemostasis in major bleeding.

• #39 Past, Present, and Future Options in the Treatment of Hemophilia A | American Society for Clinical Laboratory Science

  https://clsjournal.ascls.org/content/32/1/36

  Fresh frozen plasma, freeze dried concentrate, and cryoprecipitate work by replacing the absent or low levels of FVIII in patients. […] Recombinant-factor concentrates work by replacing FVIII levels in hemophilia patients. […] The half-life of standard FVIII products is between 812 hours. Several approaches have been used to try to increase the half-life of rFVIII to decrease the number of scheduled infusions. […] Desmopressin (DDAVP) is used intravenously or intranasally in the treatment of mild or moderate hemophilia A with a response rate of 80%90%. […] One of the treatments for patients with inhibitors is to eradicate the inhibitors by using a process known as ITI. […] Gene transfer has been successful in patients with hemophilia B; however, the large size of the FVIII-coding region has made successful gene therapy elusive for hemophilia A patients.

• #40 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. […] Replacement clotting factor can be made from donated blood. Similar products, called recombinant clotting factors, are made in a laboratory, not from human blood. […] Other therapies include: Desmopressin. In some forms of mild hemophilia, this hormone can stimulate the body to release more clotting factor. It can be injected slowly into a vein or used as a nasal spray. […] Emicizumab (Hemlibra). This is a newer drug that doesn’t include clotting factors. This drug can help prevent bleeding episodes in people with hemophilia A.

• #41 Hemophilia – Diagnosis and treatment – Mayo Clinic

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

  Clot-preserving medications. Also known as anti-fibrinolytics, these medications help prevent clots from breaking down. […] Fibrin sealants. These can be applied directly to wound sites to promote clotting and healing. Fibrin sealants are especially useful for dental work. […] Physical therapy. It can ease signs and symptoms if internal bleeding has damaged your joints. Severe damage might require surgery. […] First aid for minor cuts. Using pressure and a bandage will generally take care of the bleeding. For small areas of bleeding beneath the skin, use an ice pack. Ice pops can be used to slow down minor bleeding in the mouth.

• #42 Treatment of Hemophilia | Hemophilia | CDC

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

  Amicar is a medication that can be given through a vein or by mouth (as a pill or a liquid). It prevents blood clots from breaking down, resulting in a firmer clot, and is often used for bleeding in the mouth or after a tooth has been removed because it blocks a substance found in the saliva (spit) that breaks down clots. […] Roctavian is a gene therapy that was approved by the FDA on June 29, 2023, for the treatment of severe hemophilia A (factor VIII deficiency) in adults. […] Hemgenix is a gene therapy that was approved by the FDA on November 22, 2022, for the treatment of hemophilia B (factor IX deficiency) in adults.

• #43 Treatment of Hemophilia | Hemophilia | CDC

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

  Amicar is a medication that can be given through a vein or by mouth (as a pill or a liquid). It prevents blood clots from breaking down, resulting in a firmer clot, and is often used for bleeding in the mouth or after a tooth has been removed because it blocks a substance found in the saliva (spit) that breaks down clots. […] Roctavian is a gene therapy that was approved by the FDA on June 29, 2023, for the treatment of severe hemophilia A (factor VIII deficiency) in adults. […] Hemgenix is a gene therapy that was approved by the FDA on November 22, 2022, for the treatment of hemophilia B (factor IX deficiency) in adults.

• #44 Hemophilia – Diagnosis and treatment – Mayo Clinic

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

  Clot-preserving medications. Also known as anti-fibrinolytics, these medications help prevent clots from breaking down. […] Fibrin sealants. These can be applied directly to wound sites to promote clotting and healing. Fibrin sealants are especially useful for dental work. […] Physical therapy. It can ease signs and symptoms if internal bleeding has damaged your joints. Severe damage might require surgery. […] First aid for minor cuts. Using pressure and a bandage will generally take care of the bleeding. For small areas of bleeding beneath the skin, use an ice pack. Ice pops can be used to slow down minor bleeding in the mouth.

• #45 Hemophilia – Diagnosis and treatment – Mayo Clinic

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

  Clot-preserving medications. Also known as anti-fibrinolytics, these medications help prevent clots from breaking down. […] Fibrin sealants. These can be applied directly to wound sites to promote clotting and healing. Fibrin sealants are especially useful for dental work. […] Physical therapy. It can ease signs and symptoms if internal bleeding has damaged your joints. Severe damage might require surgery. […] First aid for minor cuts. Using pressure and a bandage will generally take care of the bleeding. For small areas of bleeding beneath the skin, use an ice pack. Ice pops can be used to slow down minor bleeding in the mouth.

• #46 New, lifechanging hemophilia treatments improve patients’ health, outlook | Discovery | UT Southwestern Medical Center

  https://utswmed.org/medblog/hemophilia-adult-treatment/

  Early trial results have been promising patients have shown clotting factor levels within a normal range for approximately three years before their levels begin to decline. […] As a comprehensive adult hemophilia treatment center, we offer patients with hemophilia care options that are not widely available. […] For example, we emphasize the benefits of specialized physical therapy, which can help: Build strong muscles to protect the joints and reduce the risk of bleeds, Increase strength and flexibility, leading to fewer accidents, Restore damaged joints or muscles after a bleed. […] Physical therapists who specialize in hemophilia can conduct a musculoskeletal ultrasound to see in real time whether there is bleeding in the joints. […] Until that day, we continue to push the boundaries of research, seeking more opportunities to help patients live full, active lives.

• #47 About Hemophilia | Hemophilia | CDC

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

  About 15% to 20% of people with inherited hemophilia A or B develop an antibody (called an inhibitor) that stops the clotting factors from being able to clot the blood and stop bleeding. Treatment of bleeding episodes becomes extremely difficult, and the cost of care for a person with an inhibitor can skyrocket because more clotting factor or a different type of clotting factor is needed.

• #48 About Hemophilia | Hemophilia | CDC

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

  About 15% to 20% of people with inherited hemophilia A or B develop an antibody (called an inhibitor) that stops the clotting factors from being able to clot the blood and stop bleeding. Treatment of bleeding episodes becomes extremely difficult, and the cost of care for a person with an inhibitor can skyrocket because more clotting factor or a different type of clotting factor is needed.

• #49 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. […] The treatment of patients with inhibitors of FVIII is difficult. Bleeding episodes in patients with low-titer inhibitors (ie, concentrations below 5 Bethesda units [BU]) occasionally can be overcome with high doses of factor VIII.

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

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

  Options in other cases include a variety of agents that bypass FVIII, such as activated FVII and emicizumab; desensitization; and immune tolerance induction. […] In patients who develop synovitis from joint bleeds, injection of radioisotopes into the joint to ablate the synovium (radiosynovectomy) can be used to decrease bleeding, slow progression of cartilage and bone damage, and prevent arthropathy. […] Increasing evidence associates hemophilia with low bone mineral density; consequently, careful assessment and management of fracture risk are recommended. Regular exercise, fall prevention strategies, and optimization of calcium and vitamin D intake are recommended, along with prophylactic factor replacement therapy in severe hemophilia. […] Desmopressin vasopressin analog, or 1-deamino-8-D-arginine vasopressin (DDAVP), is considered the treatment of choice for mild and moderate hemophilia A. It is not effective in the treatment of severe hemophilia.

• #51 Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult‐to‐treat patients

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6936224/

  Bleeding episodes can be treated with bypassing agents and potentially with novel haemostatic agents currently in development. […] The majority of patients with haemophilia A and inhibitors will become immune tolerant to FVIII following ITI, with international registries reporting success rates of 51%–79%. […] However, some patients will be difficult to tolerise and/or are unresponsive to first-line ITI, and these patients are the most complicated to treat. […] ITI may also be attempted in patients with haemophilia B and high-titre FIX inhibitors, but it is utilised less frequently than in those with haemophilia A due to a general lack of experience of its use in haemophilia B and lower overall success rates, as well as concern about anaphylactic reactions and development of nephrotic syndrome.

• #52 Haemophilia treatment today

  https://www.haemevolution.eu/patient/treatment-developments

  The SHL factor replacement products have a half-life of 10-12 hours (factor VIII) and 18-20 hours (factor IX). These time periods mean that for prophylaxis therapy, people with haemophilia A receive injections every second or third day or twice weekly for haemophilia B. […] The EHL factor replacement products have extended the half-life of factor VIII by 1,4-4-fold and factor IX by 3-5-fold, so these factors are depleted more slowly, reducing the frequency of injections e.g. for Haemophilia B patients to once every 7-21 days, or increase the trough level achieved for better protection. […] Non-factor replacement therapy aims at restoring the clotting capacity of the blood without directly replacing the missing clotting factors. Unfortunately, some individuals develop antibodies (inhibitors), or natural defences, against replacement factors, making replacement therapy ineffective. Non-replacement therapy could be a treatment option for these individuals. For haemophilia A patients, including those with inhibitors, there is currently one non-factor product available which requires infusion every 2-4 weeks.

• #53 Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult‐to‐treat patients

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6936224/

  However, there are limited clinical data and guidelines available to help guide physicians in formulating the next treatment steps in these patients. […] This review summarises currently available treatment options for patients with inhibitors, focussing on ITI regimens and those ITI strategies that may be used in difficult-to-treat patients. […] Some alternative, non-ITI approaches for inhibitor management, are also proposed. […] While the recently approved non-factor therapy, emicizumab (Hemlibra, Roche, Basel, Switzerland) provides new treatment options for patients with haemophilia A and inhibitors against FVIII, the authors do not recommend emicizumab as first-line therapy in these patients. […] The preferred management strategy for patients with haemophilia A who develop high-titre inhibitors is antibody eradication via immune tolerance induction (ITI).

• #54 Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult‐to‐treat patients

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6936224/

  The conventional methods for ITI include the Bonn high-dose regimen or variant protocols and the Van Creveld Dutch low-dose regimen, or variant protocols. […] In difficult-to-treat cases, the Malm protocol is an alternative option. […] The pivotal International Immune Tolerance (IIT) study, which randomised patients to low-dose (50 IU/kg FVIII, three times a week) or high-dose (200 IU/kg FVIII, daily) regimens, lacked statistical power to show therapeutic equivalence. […] The FVIII dose did not affect success rate at the end of the study period, although patients receiving a high dose had a significantly shorter time to negative inhibitor titre. […] In the event that ITI is successful and tolerance achieved, the authors recommend long-term continuous prophylaxis, to help maintain tolerance and to avoid rapid changes in dose.

• #55 Past, Present, and Future Options in the Treatment of Hemophilia A | American Society for Clinical Laboratory Science

  https://clsjournal.ascls.org/content/32/1/36

  Fresh frozen plasma, freeze dried concentrate, and cryoprecipitate work by replacing the absent or low levels of FVIII in patients. […] Recombinant-factor concentrates work by replacing FVIII levels in hemophilia patients. […] The half-life of standard FVIII products is between 812 hours. Several approaches have been used to try to increase the half-life of rFVIII to decrease the number of scheduled infusions. […] Desmopressin (DDAVP) is used intravenously or intranasally in the treatment of mild or moderate hemophilia A with a response rate of 80%90%. […] One of the treatments for patients with inhibitors is to eradicate the inhibitors by using a process known as ITI. […] Gene transfer has been successful in patients with hemophilia B; however, the large size of the FVIII-coding region has made successful gene therapy elusive for hemophilia A patients.

• #56 Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult‐to‐treat patients

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6936224/

  The conventional methods for ITI include the Bonn high-dose regimen or variant protocols and the Van Creveld Dutch low-dose regimen, or variant protocols. […] In difficult-to-treat cases, the Malm protocol is an alternative option. […] The pivotal International Immune Tolerance (IIT) study, which randomised patients to low-dose (50 IU/kg FVIII, three times a week) or high-dose (200 IU/kg FVIII, daily) regimens, lacked statistical power to show therapeutic equivalence. […] The FVIII dose did not affect success rate at the end of the study period, although patients receiving a high dose had a significantly shorter time to negative inhibitor titre. […] In the event that ITI is successful and tolerance achieved, the authors recommend long-term continuous prophylaxis, to help maintain tolerance and to avoid rapid changes in dose.

• #57 Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult‐to‐treat patients

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6936224/

  Bleeding episodes can be treated with bypassing agents and potentially with novel haemostatic agents currently in development. […] The majority of patients with haemophilia A and inhibitors will become immune tolerant to FVIII following ITI, with international registries reporting success rates of 51%–79%. […] However, some patients will be difficult to tolerise and/or are unresponsive to first-line ITI, and these patients are the most complicated to treat. […] ITI may also be attempted in patients with haemophilia B and high-titre FIX inhibitors, but it is utilised less frequently than in those with haemophilia A due to a general lack of experience of its use in haemophilia B and lower overall success rates, as well as concern about anaphylactic reactions and development of nephrotic syndrome.

• #58 Understanding Current Bleeding Disorder Treatments | NBDF

  https://www.bleeding.org/bleeding-disorders-a-z/treatment/current-treatments

  New treatments that use other ways of preventing bleeds are also available. These treatments are known as non-factor replacement therapies. […] It is important to discuss all treatment options with your doctor or the staff at your HTC. […] There are many new treatments for hemophilia A being developed, from gene therapy to new non-factor replacement therapies.

• #59 Exploring Future Therapies for Bleeding Disorders | NBDF

  https://www.bleeding.org/bleeding-disorders-a-z/treatment/future-therapies

  There are several new medicines, or therapies, currently being explored for the treatment of bleeding disorders. […] Most of these products are in clinical trials, which are tests that researchers do to make sure medicines work the way they should. […] Several new therapies seek to reduce bleeding episodes by restoring this balance. […] Currently, gene therapies for Hemophilia A and Hemophilia B work differently in the body and have different results. […] Gene therapy for hemophilia A and gene therapy for hemophilia B have both recently been approved by the FDA. […] Anti-TFPI is a novel treatment that seeks to reduce bleeding by decreasing (or putting the brakes) on the system that prevents us from clotting too much. […] Anti-TFPI restores hemostatic balance by blocking one of the anti-coagulants, TFPI, and preventing it from working normally.

• #60 Haemophilia treatment

  https://www.haemophilia.org.au/bleeding-disorders/haemophilia/haemophilia-treatment/

  Replacement factor therapy can be given: As prophylaxis given regularly to prevent bleeds or reduce bleeding from an injury. How often it is given is tailored to the individual and can be given at different intervals, ranging from daily to fortnightly. […] Non-factor therapies work differently to replacement factor therapy. These treatments do not replace factor VIII or factor IX but work to support the body’s capacity to clot in other ways. […] Gene therapy for haemophilia aims to provide the body with a functioning version of the factor VIII or factor IX gene so that the body can produce enough clotting factor for the blood to clot normally. […] Desmopressin (DDAVP) is a synthetic hormone that releases the body’s stored factor VIII into the bloodstream to help blood clot. […] Hormone treatment can help women who have heavy menstrual bleeding. The hormones can increase factor VIII levels. […] After treatment with a clotting factor product, some people with haemophilia may develop antibodies known as inhibitors which may mean treatment with factor replacement therapy no longer works for them or is less effective.

• #61 Hemophilia therapy: the future has begun

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7049365/

  This took place in two main ways: (i) for HA, by mimicking the coagulant activity of FVIII; and (ii) for both HA and HB, by increasing defective thrombin formation through the inhibition of the naturally occurring anticoagulants. […] For the moment, only the monoclonal antibody emicizumab that mimics FVIII activity has been licensed and marketed. […] The first vector associated with curative gene transfer in animal models of hemophilia was the adeno-associated virus (AAV), and, so far, AAV vectors are the only tools used to achieve therapeutic levels of FVIII and IX in hemophilia patients. […] The use of gene transfer was initially slower in HA, due to the much larger size of the FVIII gene that made it difficult to pack the corresponding cDNA in AAV vectors. […] However, at the moment, maximum follow up is no longer than 1.5 years, and, as for all new therapeutic developments, long-term follow up is still required to firmly consolidate the safety profile.

• #62 Hemophilia

  https://patienteducation.asgct.org/disease-treatments/hemophilia

  Hemophilia is a genetic disease that prevents blood from clotting properly leading to prolonged internal and external bleeding. […] Learn how gene therapy works to slow or stop disease progression by instructing cells to produce the missing clotting factor, along with information on approved therapies and clinical trials. […] BEQVEZ is an FDA-approved gene therapy for the treatment of adults with Hemophilia B (congenital factor IX deficiency). […] HEMGENIX is an FDA-approved gene therapy for the treatment of adults with hemophilia B (congenital Factor IX deficiency). […] ROCTAVIAN is an FDA-approved gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency). […] Gene therapy aims to be given one time with the goal of eliminating the need for recurring treatments.

• #63 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/

  New opportunities are arising in the form of non-replacement therapies, which aim to alter the hemostatic balance of a patient by enhancing thrombin generation or coagulation. […] Alongside these novel therapies, gene therapies which act to rebalance hemostasis or induce long-term replacement of the missing factor are emerging to treat hemophilia. […] The most recent and compelling development in hemophilia treatment was the approval of the first gene therapies for hemophilia B (etranacogene dezaparvovec; November 2022) and hemophilia A (valoctocogene roxaparvovec; June 2023). Both of these treatments are adeno-associated virus (AAV) vector-based gene therapies, which use a viral vector carrying the gene for the missing clotting factor in a one-time treatment. […] Despite these challenges, gene therapy is a bold new field in the treatment of genetic disorders such as hemophilia, and the recent approval of etranacogene dezaparvovec and valoctocogene roxaparvovec represent great steps forward in this area.

• #64 Hemophilia

  https://patienteducation.asgct.org/disease-treatments/hemophilia

  Hemophilia is a genetic disease that prevents blood from clotting properly leading to prolonged internal and external bleeding. […] Learn how gene therapy works to slow or stop disease progression by instructing cells to produce the missing clotting factor, along with information on approved therapies and clinical trials. […] BEQVEZ is an FDA-approved gene therapy for the treatment of adults with Hemophilia B (congenital factor IX deficiency). […] HEMGENIX is an FDA-approved gene therapy for the treatment of adults with hemophilia B (congenital Factor IX deficiency). […] ROCTAVIAN is an FDA-approved gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency). […] Gene therapy aims to be given one time with the goal of eliminating the need for recurring treatments.

• #65 Gene therapy – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-201-march-2018/gene-therapy/

  Media stories about the two gene therapy trials spoke of them as a breakthrough and a giant leap forward in the search for a cure for haemophilia. […] The two gene therapy studies that published results in December are some of the most advanced: one trial in the UK studying haemophilia A and the other in the USA, Canada and Australia studying haemophilia B. […] For most, factor levels could be increased and sustained at the mild haemophilia range or higher for at least one year. […] Prophylaxis treatment could be discontinued for all participants. […] Nearly all participants had no further bleeding episodes. […] The haemophilia A gene therapy study took place across multiple centres in the UK and was sponsored by BioMarin Pharmaceutical. […] The haemophilia B gene therapy trial was a collaboration between the team led by Prof. John Rasko at the Royal Prince Alfred Hospital in Sydney and multiple centres in the USA and Canada, and was sponsored by Spark Therapeutics.

• #66 Exploring Future Therapies for Bleeding Disorders | NBDF

  https://www.bleeding.org/bleeding-disorders-a-z/treatment/future-therapies

  There are several new medicines, or therapies, currently being explored for the treatment of bleeding disorders. […] Most of these products are in clinical trials, which are tests that researchers do to make sure medicines work the way they should. […] Several new therapies seek to reduce bleeding episodes by restoring this balance. […] Currently, gene therapies for Hemophilia A and Hemophilia B work differently in the body and have different results. […] Gene therapy for hemophilia A and gene therapy for hemophilia B have both recently been approved by the FDA. […] Anti-TFPI is a novel treatment that seeks to reduce bleeding by decreasing (or putting the brakes) on the system that prevents us from clotting too much. […] Anti-TFPI restores hemostatic balance by blocking one of the anti-coagulants, TFPI, and preventing it from working normally.

• #67 Exploring Future Therapies for Bleeding Disorders | NBDF

  https://www.bleeding.org/bleeding-disorders-a-z/treatment/future-therapies

  RNA interference (RNAi) therapy targets an anti-coagulant called antithrombin. […] It is a novel therapy that seeks to restore hemostatic balance by decreasing antithrombin which allows the formation of enough thrombin, a coagulant, to prevent bleeding. […] The best person to discuss new therapies to treat bleeding disorders is your healthcare provider at your hemophilia treatment center.

• #68 Exploring Future Therapies for Bleeding Disorders | NBDF

  https://www.bleeding.org/bleeding-disorders-a-z/treatment/future-therapies

  There are several new medicines, or therapies, currently being explored for the treatment of bleeding disorders. […] Most of these products are in clinical trials, which are tests that researchers do to make sure medicines work the way they should. […] Several new therapies seek to reduce bleeding episodes by restoring this balance. […] Currently, gene therapies for Hemophilia A and Hemophilia B work differently in the body and have different results. […] Gene therapy for hemophilia A and gene therapy for hemophilia B have both recently been approved by the FDA. […] Anti-TFPI is a novel treatment that seeks to reduce bleeding by decreasing (or putting the brakes) on the system that prevents us from clotting too much. […] Anti-TFPI restores hemostatic balance by blocking one of the anti-coagulants, TFPI, and preventing it from working normally.

• #69 Exploring Future Therapies for Bleeding Disorders | NBDF

  https://www.bleeding.org/bleeding-disorders-a-z/treatment/future-therapies

  RNA interference (RNAi) therapy targets an anti-coagulant called antithrombin. […] It is a novel therapy that seeks to restore hemostatic balance by decreasing antithrombin which allows the formation of enough thrombin, a coagulant, to prevent bleeding. […] The best person to discuss new therapies to treat bleeding disorders is your healthcare provider at your hemophilia treatment center.

• #70 Advances in the management of haemophilia: emerging treatments and their mechanisms | Journal of Biomedical Science | Full Text

  https://jbiomedsci.biomedcentral.com/articles/10.1186/s12929-021-00760-4

  Emicizumab, a recombinant humanised bispecific IgG antibody, mimics the cofactor function of the missing FVIII in HA. It simultaneously binds activated FIX (FIXa) and factor X (FX), bringing them into spatial proximity to promote FIXa-catalysed FX activation, thereby restoring haemostasis. […] The unique molecular structure of emicizumab means it is not subject to the same physiologic mechanisms that eliminate FVIII from circulation, hence it persists in circulation for much longer compared to FVIII. It has a long half-life of around 45 weeks, which supports dosing regimens once weekly, every 2 weeks or even monthly. […] Fitusiran (ALN-AT3), a novel therapy applicable to both HA and HB, consists of the amino acid, N-Acetylgalactosamine (GalNAc), the ligand of the hepatic asialoglycoprotein receptors, conjugated to a synthetic siRNA. Administered subcutaneously, it targets and degrades a region of the SERPINC1 gene mRNA, preventing antithrombin production and enhancing thrombin generation.

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

  https://emedicine.medscape.com/article/779434-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. […] 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. […] Gene therapy offers the potential for a definitive cure and has entered clinical practice, with FDA approval of the first product in 2022.

• #72 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). […] Todays approval of Hympavzi provides patients with hemophilia a new treatment option that is the first of its kind to work by targeting a protein in the blood clotting process, said Ann Farrell, M.D., Director of the Division of Non-Malignant Hematology in the FDAs Center for Drug Evaluation and Research. This new type of treatment underscores the FDAs commitment to advance the development of innovative, safe and effective therapies. […] 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.

• #73 New, lifechanging hemophilia treatments improve patients’ health, outlook | Discovery | UT Southwestern Medical Center

  https://utswmed.org/medblog/hemophilia-adult-treatment/

  Instead of having to find a vein for multiple IV infusions a week, emicizumab is given through an injection under the skin once a week, once every two weeks, or once a month. […] This has made a huge difference in terms of the burden of the treatment on our patients. […] However, we expect a new once-a-month subcutaneous hemophilia injection, Fitusiran, to be available soon for patients with hemophilia A and those who are missing clotting factor IX (hemophilia B). […] While several types of gene therapies are being studied in clinical trials, gene transfer via adeno-associated virus (AAV) vector-based gene therapy is furthest along the pipeline. […] AAV vector-based gene therapy uses a modified virus to deliver functional copies of factor VIII or factor IX genes to cells in the liver, empowering the body to make its own clotting factor.

• #74 Haemophilia treatment

  https://www.haemophilia.org.au/bleeding-disorders/haemophilia/haemophilia-treatment/

  Treatment and care for people with haemophilia involves a team of specialist health professionals to cover the range of their care needs. This is known as comprehensive care. […] Comprehensive care is coordinated by a Haemophilia Treatment Centre. […] Each person affected by haemophilia will have their own treatment plan, which is developed with them individually and reviewed regularly/as needed with their haematologist (blood diseases specialist doctor) or haemophilia nurse practitioner. It may include a plan for regular treatment and for injuries or surgery. […] There are a variety of treatment products used to treat haemophilia. It is important that people with haemophilia discuss these treatment options with their Haemophilia Treatment Centre. […] Clotting factor concentrates replace the missing clotting factor in the blood and are called replacement factor therapy.

• #75 Treatment of Hemophilia | Hemophilia | CDC

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

  Good quality medical care from healthcare providers who know a lot about the disorder can help people with hemophilia prevent some serious problems. This type of specialty care is at a comprehensive hemophilia treatment center (HTC). An HTC provides patients with the care and education to address all issues related to the disorder. […] The two main types of clotting factor concentrates available are plasma-derived factor concentrates and recombinant factor concentrates. […] Hemlibra works by replacing the function of factor VIII, rather than replacing the missing clotting factor VIII directly. It can be used to either prevent or reduce the frequency of bleeding episodes in people with hemophilia A. […] DDAVP and Stimate are medications that are similar to a hormone that occurs naturally in the body. The medications release factor VIII from where it is stored in the body tissues.

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

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

  The treatment of hemophilia may involve prophylaxis, management of bleeding episodes, treatment of factor VIII (FVIII) inhibitors, and treatment and rehabilitation of hemophilia synovitis. Use of factor replacement products and other medications, including pain medications, is typically required. […] Treatment of patients with hemophilia ideally should be provided through a comprehensive hemophilia care center. These centers, which are found in many US cities, follow a multidisciplinary approach, with specialists in hematology, orthopedics, dentistry, and surgery; nurses; physiotherapists; social workers; and related allied health professionals. Patients treated at comprehensive care clinics have been shown to have better access to care, less morbidity, and better overall outcome. […] Ambulatory replacement therapy for bleeding episodes is essential for preventing chronic arthropathy and deformities. Home treatment and infusion by the family or patient is possible in most cases. Prompt and appropriate treatment of hemorrhage is important to prevent long-term complications and disability.

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

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

  The treatment of hemophilia may involve management of hemostasis, management of bleeding episodes, use of factor replacement products and medications, treatment of patients with factor inhibitors, and treatment and rehabilitation of patients with hemophilic synovitis. […] Treatment of patients with hemophilia ideally should be provided through a comprehensive hemophilia care center. These centers follow a multidisciplinary approach, with specialists in hematology, orthopedics, dentistry, and surgery; nurses; physiotherapists; social workers; and related allied health professionals. Patients treated at comprehensive care clinics have been shown to have better access to care, less morbidity, and better overall outcome. […] Ambulatory replacement therapy for bleeding episodes is essential for preventing chronic arthropathy and deformities. Home treatment and infusion by the family or patient is possible in most cases. Prompt and appropriate treatment of hemorrhage is important to prevent long-term complications and disability.

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

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

  The treatment of hemophilia may involve prophylaxis, management of bleeding episodes, treatment of factor VIII (FVIII) inhibitors, and treatment and rehabilitation of hemophilia synovitis. Use of factor replacement products and other medications, including pain medications, is typically required. […] Treatment of patients with hemophilia ideally should be provided through a comprehensive hemophilia care center. These centers, which are found in many US cities, follow a multidisciplinary approach, with specialists in hematology, orthopedics, dentistry, and surgery; nurses; physiotherapists; social workers; and related allied health professionals. Patients treated at comprehensive care clinics have been shown to have better access to care, less morbidity, and better overall outcome. […] Ambulatory replacement therapy for bleeding episodes is essential for preventing chronic arthropathy and deformities. Home treatment and infusion by the family or patient is possible in most cases. Prompt and appropriate treatment of hemorrhage is important to prevent long-term complications and disability.

• #79 Haemophilia treatment

  https://www.haemophilia.org.au/bleeding-disorders/haemophilia/haemophilia-treatment/

  Treatment and care for people with haemophilia involves a team of specialist health professionals to cover the range of their care needs. This is known as comprehensive care. […] Comprehensive care is coordinated by a Haemophilia Treatment Centre. […] Each person affected by haemophilia will have their own treatment plan, which is developed with them individually and reviewed regularly/as needed with their haematologist (blood diseases specialist doctor) or haemophilia nurse practitioner. It may include a plan for regular treatment and for injuries or surgery. […] There are a variety of treatment products used to treat haemophilia. It is important that people with haemophilia discuss these treatment options with their Haemophilia Treatment Centre. […] Clotting factor concentrates replace the missing clotting factor in the blood and are called replacement factor therapy.

• #80 How Is Hemophilia Treated? | Hematology-Oncology Associates of CNY

  https://www.hoacny.com/patient-resources/blood-disorders/what-hemophilia/how-hemophilia-treated

  Antibodies can destroy the clotting factor before it has a chance to work. This is a very serious problem. It prevents the main treatment for hemophilia (replacement therapy) from working. […] You can do both preventive (ongoing) and demand (as-needed) replacement therapy at home. Many people learn to do the infusions at home for their child or for themselves. Home treatment has several advantages: You or your child can get quicker treatment when bleeding happens. Early treatment lowers the risk of complications. […] Discuss options for home treatment with your doctor or your child’s doctor. […] Desmopressin (DDAVP) is a man-made hormone used to treat people who have mild hemophilia A. […] Antifibrinolytic medicines (including tranexamic acid and epsilon aminocaproic acid) may be used with replacement therapy.

• #81 Treating Hemophilia B | Bleeding Disorders

  https://www.bleedingdisorders.com/hemophilia-b/treatment

  Fewer visits to the healthcare provider (HCP) or emergency room are needed. Home treatment costs less than treatment in a medical care setting. […] Home treatment helps children accept treatment and take responsibility for their own health. […] Today, clotting factor IX concentrates can be made without human plasma. Infusions are called recombinant clotting factors. […] In the past 25+ years, significant advances have been made in the manufacturing of these recombinant clotting factors. […] Recombinant technology has helped produce factor concentrates that avoid the risk of human-viral contamination. […] The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) encourages the use of recombinant clotting factor products. Your HCP or your hemophilia treatment center (HTC) will help you decide which is right for you. […] Factor treatment has to be personalized to your lifestyle.

• #82 How Is Hemophilia Treated? | Hematology-Oncology Associates of CNY

  https://www.hoacny.com/patient-resources/blood-disorders/what-hemophilia/how-hemophilia-treated

  Antibodies can destroy the clotting factor before it has a chance to work. This is a very serious problem. It prevents the main treatment for hemophilia (replacement therapy) from working. […] You can do both preventive (ongoing) and demand (as-needed) replacement therapy at home. Many people learn to do the infusions at home for their child or for themselves. Home treatment has several advantages: You or your child can get quicker treatment when bleeding happens. Early treatment lowers the risk of complications. […] Discuss options for home treatment with your doctor or your child’s doctor. […] Desmopressin (DDAVP) is a man-made hormone used to treat people who have mild hemophilia A. […] Antifibrinolytic medicines (including tranexamic acid and epsilon aminocaproic acid) may be used with replacement therapy.

• #83 Treating Hemophilia B | Bleeding Disorders

  https://www.bleedingdisorders.com/hemophilia-b/treatment

  Fewer visits to the healthcare provider (HCP) or emergency room are needed. Home treatment costs less than treatment in a medical care setting. […] Home treatment helps children accept treatment and take responsibility for their own health. […] Today, clotting factor IX concentrates can be made without human plasma. Infusions are called recombinant clotting factors. […] In the past 25+ years, significant advances have been made in the manufacturing of these recombinant clotting factors. […] Recombinant technology has helped produce factor concentrates that avoid the risk of human-viral contamination. […] The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) encourages the use of recombinant clotting factor products. Your HCP or your hemophilia treatment center (HTC) will help you decide which is right for you. […] Factor treatment has to be personalized to your lifestyle.

• #84 Treating Hemophilia B | Bleeding Disorders

  https://www.bleedingdisorders.com/hemophilia-b/treatment

  Fewer visits to the healthcare provider (HCP) or emergency room are needed. Home treatment costs less than treatment in a medical care setting. […] Home treatment helps children accept treatment and take responsibility for their own health. […] Today, clotting factor IX concentrates can be made without human plasma. Infusions are called recombinant clotting factors. […] In the past 25+ years, significant advances have been made in the manufacturing of these recombinant clotting factors. […] Recombinant technology has helped produce factor concentrates that avoid the risk of human-viral contamination. […] The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) encourages the use of recombinant clotting factor products. Your HCP or your hemophilia treatment center (HTC) will help you decide which is right for you. […] Factor treatment has to be personalized to your lifestyle.

• #85 Treating Hemophilia B | Bleeding Disorders

  https://www.bleedingdisorders.com/hemophilia-b/treatment

  Fewer visits to the healthcare provider (HCP) or emergency room are needed. Home treatment costs less than treatment in a medical care setting. […] Home treatment helps children accept treatment and take responsibility for their own health. […] Today, clotting factor IX concentrates can be made without human plasma. Infusions are called recombinant clotting factors. […] In the past 25+ years, significant advances have been made in the manufacturing of these recombinant clotting factors. […] Recombinant technology has helped produce factor concentrates that avoid the risk of human-viral contamination. […] The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) encourages the use of recombinant clotting factor products. Your HCP or your hemophilia treatment center (HTC) will help you decide which is right for you. […] Factor treatment has to be personalized to your lifestyle.

• #86 Treatment of Hemophilia | Hemophilia | CDC

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

  If you have hemophilia, you need treatment so your blood can clot. […] Treatment products have different ways of helping blood clot. […] Hemophilia treatment centers specialize in caring for people with bleeding disorders. […] 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. […] For hemophilia A, another treatment method is a non-factor product that stands in for, or replaces the function of, the missing factor VIII. This treatment is given by injection under the skin that can be given by people with hemophilia themselves. […] Healthcare providers typically prescribe treatment products for episodic care or prophylactic care. Episodic care is used to stop a patients bleeding episodes; 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. Giving factor treatment products at home means that bleeds can be treated more quickly, resulting in less serious bleeding and fewer side effects.

• #87 Haemophilia Treatment | Medica Superspecialty Hospital

  https://medicahospitals.in/blog/haemophilia-symptoms-diagnosis-and-treatment/

  Haemophilia is a bleeding disorder in which the blood does not clot properly. […] The ideal way to treat haemophilia is to replace the missing blood clotting factor, so that the blood can clot properly. This is done by injecting treatment products, known as clotting factor concentrates, into an individuals vein. […] Clinicians typically prescribe treatment products for episodic care or prophylactic care. Episodic care is used to stop an individuals bleeding episodes; prophylactic care is used to prevent bleeding episodes from occurring. […] Today, its possible for people with haemophilia, and their families, to learn how to give their own clotting factor treatment products at home. Giving factor treatment products at home indicates that bleeds can be treated faster, resulting in comparatively less serious bleeding and fewer side effects.

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

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

  Patients can be treated with prophylaxis or with intermittent, on-demand therapy for bleeding events. 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, resulting in decreased overall joint deterioration and improved 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 A without inhibitors be treated prophylactically rather than through episodic management of bleeding events.

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

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

  Patients may be treated with prophylaxis or with intermittent, on-demand therapy for bleeding events. 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.

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

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

  Options in other cases include a variety of agents that bypass FVIII, such as activated FVII and emicizumab; desensitization; and immune tolerance induction. […] In patients who develop synovitis from joint bleeds, injection of radioisotopes into the joint to ablate the synovium (radiosynovectomy) can be used to decrease bleeding, slow progression of cartilage and bone damage, and prevent arthropathy. […] Increasing evidence associates hemophilia with low bone mineral density; consequently, careful assessment and management of fracture risk are recommended. Regular exercise, fall prevention strategies, and optimization of calcium and vitamin D intake are recommended, along with prophylactic factor replacement therapy in severe hemophilia. […] Desmopressin vasopressin analog, or 1-deamino-8-D-arginine vasopressin (DDAVP), is considered the treatment of choice for mild and moderate hemophilia A. It is not effective in the treatment of severe hemophilia.

• #91 My nephew has hemophilia. Are there any new treatments?

  https://www.icliniq.com/qa/hemophilia/what-are-the-new-treatment-options-for-hemophilia

  If his bleeding episodes are frequent, he should continue with prophylactic treatment and regular doses of clotting factor before activities like exercise or sports to help prevent spontaneous bleeds. […] For physical activity, encourage him to take part in low-impact activities like swimming, cycling, or walking. These activities are safer and help maintain cardiovascular fitness without putting him at high risk for injury. […] Higher-risk sports like basketball or soccer can be safer with protective gear, such as knee pads, elbow pads, and helmets, which can help reduce the risk of injury if he falls. […] It is also important to educate him and the people around him like friends, teachers, and coaches about his condition. This way, they will better understand her limits and help him stay safe.

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

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

  Options in other cases include a variety of agents that bypass FVIII, such as activated FVII and emicizumab; desensitization; and immune tolerance induction. […] In patients who develop synovitis from joint bleeds, injection of radioisotopes into the joint to ablate the synovium (radiosynovectomy) can be used to decrease bleeding, slow progression of cartilage and bone damage, and prevent arthropathy. […] Increasing evidence associates hemophilia with low bone mineral density; consequently, careful assessment and management of fracture risk are recommended. Regular exercise, fall prevention strategies, and optimization of calcium and vitamin D intake are recommended, along with prophylactic factor replacement therapy in severe hemophilia. […] Desmopressin vasopressin analog, or 1-deamino-8-D-arginine vasopressin (DDAVP), is considered the treatment of choice for mild and moderate hemophilia A. It is not effective in the treatment of severe hemophilia.

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

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

  Options in other cases include a variety of agents that bypass FVIII, such as activated FVII and emicizumab; desensitization; and immune tolerance induction. […] In patients who develop synovitis from joint bleeds, injection of radioisotopes into the joint to ablate the synovium (radiosynovectomy) can be used to decrease bleeding, slow progression of cartilage and bone damage, and prevent arthropathy. […] Increasing evidence associates hemophilia with low bone mineral density; consequently, careful assessment and management of fracture risk are recommended. Regular exercise, fall prevention strategies, and optimization of calcium and vitamin D intake are recommended, along with prophylactic factor replacement therapy in severe hemophilia. […] Desmopressin vasopressin analog, or 1-deamino-8-D-arginine vasopressin (DDAVP), is considered the treatment of choice for mild and moderate hemophilia A. It is not effective in the treatment of severe hemophilia.

• #94 Get Hemophilia Treatment | Cleveland Clinic

  https://my.clevelandclinic.org/services/hemophilia-treatment

  If you have severe hemophilia, you may also get factor infusions to prevent frequent or uncontrolled bleeding. This type of treatment is called prophylactic therapy. […] We help you manage your condition and learn what things you should avoid, like activities where you can get bumped, hit or may fall, like football, hockey, boxing, wrestling, rugby or skateboarding. […] We have the answers and personalized care you need. Were here to help you manage this rare blood disorder by reducing your symptoms and finding ways to safely live life to the fullest.

• #95 My nephew has hemophilia. Are there any new treatments?

  https://www.icliniq.com/qa/hemophilia/what-are-the-new-treatment-options-for-hemophilia

  If his bleeding episodes are frequent, he should continue with prophylactic treatment and regular doses of clotting factor before activities like exercise or sports to help prevent spontaneous bleeds. […] For physical activity, encourage him to take part in low-impact activities like swimming, cycling, or walking. These activities are safer and help maintain cardiovascular fitness without putting him at high risk for injury. […] Higher-risk sports like basketball or soccer can be safer with protective gear, such as knee pads, elbow pads, and helmets, which can help reduce the risk of injury if he falls. […] It is also important to educate him and the people around him like friends, teachers, and coaches about his condition. This way, they will better understand her limits and help him stay safe.

• #96 Hemophilia therapy: the future has begun

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7049365/

  The success story of hemophilia care first began in the 1970s, when the availability of plasma-derived concentrates of coagulation factor VIII (FVIII) and factor IX (FIX) provided efficacious treatment of bleeding in patients with hemophilia A and B. […] This positive scenario was consolidated in terms of greater safety and availability in the 1990s, when the first recombinant coagulation factors were produced. […] This meant that, instead of only treating episodic bleeding events, prophylaxis regimens could be implemented as a preventive measure. […] Following the demonstration of its superiority in the frame of two randomized clinical trials, prophylaxis became evidence-based standard of care. […] In high-income countries, these achievements have led to a patients life expectancy being extended to close to that of the general male population.

• #97 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 […] VJHemOnc is excited to present the latest developments in the treatment of hemophilia […] 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).

• #98 Hemophilia Treatment Advances: From Factor Replacement to Gene Therapy – Green Bay Oncology

  https://gboncology.com/hemophilia-treatment-advances-from-factor-replacement-to-gene-therapy/

  Were in a remarkable era for hemophilia treatment, with options available today that were unimaginable just a decade ago. […] A condition that once significantly limited life expectancy and quality of life is now increasingly manageable, thanks to groundbreaking treatment advancements including extended half-life factors, non-factor therapies, and gene therapy. […] Historically, hemophilia treatment focused on replacing the missing clotting factor through infusions. […] The development of extended half-life factor replacement therapies marked a significant advancement in hemophilia treatment. […] Next came a new class of drugs known as non-factor therapies, which act differently from traditional factor replacement. […] The most revolutionary advancement in hemophilia treatment may be gene therapy.

• #99 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/

  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.

• #100 Hemophilia: Our Approach to New Therapeutics

  https://www.sanofi.com/en/magazine/our-science/science-behind-sanofis-hemophilia-pipeline

  People living with hemophilia are looking for treatments that last longer and make life more predictable and manageable. […] Thats why scientists are investigating three different approaches to preventing bleeds in people with missing or insufficient blood-clotting factors: […] In clinical studies, were investigating a new class of factor VIII replacement therapy. It’s designed to sustain high protection from bleeds in people with hemophilia A, with factor levels in the normal to near-normal range for most of the week. […] Our goal is to help people live more active lives and think about their treatments less. […] Were studying a treatment thats based on small interfering RNA (siRNA). Its designed to rebalance the missing blood-clotting factors in people with hemophilia A and B, with or without inhibitors (antibodies that attack replacement factors), potentially offering prophylaxis with as few as 6 injections per year.

• #101 Gene therapy – Haemophilia Foundation Australia

  https://www.haemophilia.org.au/national-haemophilia/no-201-march-2018/gene-therapy/

  Professor Rasko sees this as a major step in haemophilia treatment. […] We now know how to beat the immune response to achieve what may be a permanent cure. […] There is still a long way to travel with both of these gene therapy trials. […] Before these treatments can be made widely available, there will need to be studies of much larger groups of people with haemophilia who, along with these participants, will need to be monitored for at least 15 years to confirm the results and check for other complications. […] A next step for the haemophilia A AAV5 study is to trial a high dose in a small number of people who are AAV positive to see what the outcome is. […] The real potential of a cure with safe and effective gene therapy is very exciting for people living with this chronic condition, and for their families. […] For people with haemophilia in Australia, these are exciting times, with a range of new ground-breaking haemophilia treatments coming on to the market as well as the first indications of success in these experimental gene therapy trials.

• #102 Hemophilia

  https://patienteducation.asgct.org/disease-treatments/hemophilia

  Hemophilia is a genetic disease that prevents blood from clotting properly leading to prolonged internal and external bleeding. […] Learn how gene therapy works to slow or stop disease progression by instructing cells to produce the missing clotting factor, along with information on approved therapies and clinical trials. […] BEQVEZ is an FDA-approved gene therapy for the treatment of adults with Hemophilia B (congenital factor IX deficiency). […] HEMGENIX is an FDA-approved gene therapy for the treatment of adults with hemophilia B (congenital Factor IX deficiency). […] ROCTAVIAN is an FDA-approved gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency). […] Gene therapy aims to be given one time with the goal of eliminating the need for recurring treatments.

• #103 Hemophilia therapy: the future has begun

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7049365/

  The success story of hemophilia care first began in the 1970s, when the availability of plasma-derived concentrates of coagulation factor VIII (FVIII) and factor IX (FIX) provided efficacious treatment of bleeding in patients with hemophilia A and B. […] This positive scenario was consolidated in terms of greater safety and availability in the 1990s, when the first recombinant coagulation factors were produced. […] This meant that, instead of only treating episodic bleeding events, prophylaxis regimens could be implemented as a preventive measure. […] Following the demonstration of its superiority in the frame of two randomized clinical trials, prophylaxis became evidence-based standard of care. […] In high-income countries, these achievements have led to a patients life expectancy being extended to close to that of the general male population.

• #104 What Is Hemophilia?

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

  Hemophilia happens when you don’t have the normal amount of clotting factors. Healthcare providers treat hemophilia by replacing missing clotting factors. […] Healthcare providers treat this condition by replacing the missing clotting factors. There isn’t a cure for hemophilia, but people who receive treatment generally live about as long as people who don’t have hemophilia. 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). In replacement therapy, you receive human plasma concentrates or lab-made (recombinant) clotting factors. In general, only people with severe hemophilia need regular replacement therapy. People with mild or moderate hemophilia who need surgery may receive replacement therapy.

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