Medication
Treatment
- Hemophilia A is treated with prescription hormone, desmopressin. This can be administered by being injected into a vein or as a nasal spray. ...
- Hemophilia B is treated by infusing donor clotting factors into the blood of someone suffering from it. ...
- Hemophilia C can be treated by plasma infusion which helps in stopping hemorrhaging.
Therapy
The main treatment for severe hemophilia involves receiving replacement of the specific clotting factor that you need through a tube placed in a vein. This replacement therapy can be given to combat a bleeding episode that's in progress. It can also be administered on a regular schedule at home to help prevent bleeding episodes.
Self-care
Secondary prevention Prophylaxis is indicated in most patients with severe hemophilia A or B. It is defined as the regular intravenous factor replacement given for at least 45 weeks/year in anticipation of, and to prevent, bleeding. There are different types of prophylaxis:
Nutrition
The primary treatment for hemophilia is replacement therapy that supplies the clotting factors to hemophilia patients who lack them. Other approved treatments to control bleeding episodes include antifibrinolytics, bypassing agents, and other prophylactic treatments designed to prevent bleeds.
What are the ways to prevent hemophilia?
Can hemophilia be treated and how to treat it?
What is the prevention for hemophilia?
Are there any treatments or medications used for hemophilia?
What is the treatment for hemophilia B?
The most common treatment for hemophilia B is called replacement therapy. Concentrates of clotting factor IX are the foundation of this treatment for hemophilia B and are administered as an infusion to help replace the clotting factor that's missing or low in the blood.
Which of the following is used in the treatment of hemophilia A?
Hemlibra® (also known as ACE 910 or emicizumab) It can be used to either prevent or reduce the frequency of bleeding episodes in people with hemophilia A. This treatment product can be given by injection under the skin.
What is hemophilia B with inhibitors?
People with hemophilia A or B can develop inhibitors, which prevent their factor VIII or IX replacement treatment from working to form a clot to stop bleeding. People with hemophilia lack certain clotting factor proteins necessary to control bleeds.
What is Haemophilia B vaccine?
Preventative treatment for people with haemophilia B involves regular injections of a medicine called nonacog alfa (BeneFix). Read about nonacog alfa (BeneFix) on the European Medicines Agency's website. This is an engineered version of clotting factor IX (9), which people with haemophilia B do not have enough of.
What is the most widely accepted treatment used in hemophilia A?
Some individuals with mild hemophilia A may be treated with desmopressin (DDAVP), a synthetic agent that is a derivative of the hormone vasopressin. Desmopressin raises the plasma levels of factor VIII. Desmopressin may be administered intravenously or through a nasal spray.
How is hemophilia treated with gene therapy?
Gene therapy offers the potential for a cure for patients with hemophilia by establishing continuous endogenous expression of factor VIII or factor IX (FIX) following transfer of a functional gene to replace the hemophilic patient's own defective gene.
How is hemophilia inhibitor treated?
About Inhibitors People with hemophilia, and many with VWD type 3, use treatment products called clotting factor concentrates (“factor”). These treatment products improve blood clotting, and they are used to stop or prevent a bleeding episode.
What are hemophilia inhibitors?
An inhibitor is an immune system response to infused clotting factor concentrates, which renders standard replacement therapy ineffective. An estimated 1/3 to 1/5 of people with severe hemophilia A and 1% to 4% of those with severe hemophilia B may develop an inhibitor.
What Is factor VIII inhibitor?
FACTOR VIII INHIBITORS. The most common autoantibodies that affect clotting factor activity and lead to a bleeding disorder are directed against, and interfere with, the activity of factor VIII, a condition also called acquired hemophilia A [2-7].
What is Hib vaccine hepatitis B?
Haemophilus b conjugate vaccine and hepatitis B vaccine is a combination immunizing agent that is used to prevent infection caused by the Haemophilus influenzae type b (Hib) bacteria and hepatitis B virus.
What is the cause of hemophilia B?
Medications for Hemophilia B. Hemophilia B is a hereditary blood coagulation disorder. It is caused by a deficiency of a blood plasma protein called factor IX. Without enough factor IX, the blood cannot clot properly to control bleeding.
Is abuse a low potential for abuse relative to those in Schedule 4?
Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
Home Remedies and Lifestyle
There is no cure for hemophilia B, but it’s possible for you and your family to learn how to prevent injury and administer your own clotting factor treatments at home.
Over-the-Counter (OTC) Therapies
Bleeding into the joints and bruising can be painful. Unfortunately, common OTC pain medications like aspirin and Advil or Motrin IB can interfere with the body’s ability to clot blood. Therefore, you may want to substitute these medications with Tylenol ( acetaminophen) for pain relief. 2
Prescriptions
While there is no cure for hemophilia B, the disease can be treated over the long term with prescription replacements for the clotting factor IX that is missing in children and adults with this condition. Other medications may also be prescribed to help promote blood clotting. Treatments include:
Specialist-Driven Procedures
Surgery is not a common treatment for hemophilia B, but sometimes surgery or other treatments such as blood transfusions for heavy blood loss or intravenous administration of FIX may be given at a comprehensive hemophilia treatment center (HTC).
Complementary and Alternative Medicine (CAM)
There are no CAM therapies approved for hemophilia B, and some therapies can put you at greater risk for a bleeding episode. It's important to discuss any CAM treatment you are considering with a hematologist (blood disorder specialist) to make sure that it's safe for your condition.
Summary
The mainstay treatment for hemophilia B is the prophylactic (preventive) administration of clotting factor IX, but specific treatments depend on the severity of your hemophilia and the need to manage your acute needs at the time. A personalized treatment plan that is tailored to you can alleviate symptoms and lower your risk of complications.
A Word From Verywell
Comprehensive treatment of your hemophilia usually requires seeing multiple specialists and other healthcare providers. You and your caregivers should have access to additional information regarding new treatment options, with a specific emphasis on providing personalized health education adapted to the present challenges that you may be facing.
How to treat hemophilia B?
The current, most-used treatment for hemophilia B is factor replacement therapy. This is done by infusing (giving the medication into a vein) a FIX productinto the affected person. When you infuse FIX concentrate, the FIX is immediately available in the bloodstream, and your body can use it to stop or prevent the bleeding. Some bleeding may be successfully treated with amino caproic acid, often most useful for mouth bleeds, or tranexamic acid, also useful for excessive menstrual bleeding. Both of these medications can be given orally.
What is the Hemophilia Federation of America?
Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
When to start primary bleed treatment?
Primary – This type of treatment is usually started in young children at or before the first joint bleed to reduce or prevent joint disease and is continued indefinitely.
What is a butterfly needle used for?
Peripheral venous access, where a butterfly needle is inserted directly into a vein and is used to infuse FIX into the bloodstream, is commonly used by those who infuse at home but do not have a venous access device, such as a port, PICC line, or BROVIAC®.Below are some considerations regarding this type of access:
What is the main aim of hemophilia treatment?
The main aim of hemophilia treatment is to prevent life-threatening bleeding and/or treat muscle and joint bleeding. Early, treatment was limited to on-demand therapy, ie, the infusion of concentrate after the occurrence of the bleeding, also allowing the home therapy a prompt and successful treatment.43
When was hemophilia first treated?
After assessing that hemophilia was due to the lack of a plasma clotting factor,9the disease’s first treatment was introduced in 1948 by the infusion of plasma from healthy blood donors.10In 1958, Nilsson 1M and Blomback B in Sweden successfully treated HA patients with a globulin fraction of human plasma purified by the Cohn’s fractionation method and contained in the fraction 1-0.11At about the same time, in 1964, a new era of hemophilia therapy started after the discovery of cryoprecipitate from the plasma of a single blood donor.12,13Unfortunately, cryoprecipitate’s specific activity was relatively low (FVIII:C 0.2 IU/mg/mL).13,14The need for production and storage at −20°C in plastic bags made this approach to hemophilia therapy cumbersome and inconvenient. Furthermore, each cryoprecipitate bag contained only about 100 IU of FVIIl. A hypothetical dose of 20–30 IU/kg required the administration of about 14 or 21 bags to a PWH of 70 kg body weight. The pharmaceutical industry soon began producing lyophilized FVIII concentrates, starting from a large pool of plasma. Again, the first step was the cryo-precipitation of the plasma, but purification methods allowed the production process to achieve a remarkably high specific activity, mainly through immunoaffinity chromatography with monoclonal antibodies specific for FVIll. Unfortunately, the concentrates from large plasma pools and the single donor cryoprecipitates were contaminated by the hepatitis viruses15even though the infection risk was lower for cryoprecipitates.16Later, HIV transmission occurred in 75% of HA patients treated with commercial clotting factor concentrates17but only in 20% of those treated with single donor cryoprecipitates,18possibly since the donors were known to the patient, typically a parent. HIV transmission occurred in more than 50% of the PWH in the United States;19In Italy, probably due to the large availability of the first Prothrombin Complex Concentrates,20the prevalence of HIV infection was 47.1% in people with hemophilia B but 26.8% among those with hemophilia A.21Before the end of the last millennium, three significant advances in the manufacturing process of concentrates impacted the viral safety of products used for replacement therapy:
How much factor should I use for hemophilia?
The observation that in hemophilia patients moderate (FVIII/IX: 1–5 IU/dL) or mild disease (FVIII/IX: 5–40 IU/dL), bleeding was uncommon or absent suggested that increasing the factor level with an adequate amount of FVIII/IX concentrate every two or three other days to maintain the FVIII/FIX plasma concentration above 1IU/dL might prevent bleeding. According to a study conducted in the Netherlands, only a baseline FVIII of at least 12 IU/dL was able to entirely abolish the risk of joint bleeding. Another study from the US examined data collected over an 11-year period from males with non-severe hemophilia A or B without inhibitors who were treated on-demand with factor therapy. These authors showed that levels of 20% may be required to prevent all joint bleeding.47Another study from the Netherlands showed that PWH who have a baseline factor activity of at least 5 IU/dL experience one episode of joint bleeding each year while those with a baseline level of 3 IU/dL experience two episodes yearly.48Unfortunately, even a single episode of joint bleeding may trigger hemophilia arthropathy.49
How does the coagulation system work?
The coagulation system is a complex mechanism in which procoagulant and anticoagulant proteins determine the correct coagulation balance. The hemostatic status could be altered, increasing a bleeding or thrombotic tendency when changes affect the coagulation cascade’s balance. The bleeding phenotype of hemophilia patients is determined by several factors that influence the natural hemostatic balance83,84based on the equilibrium between natural procoagulant and anticoagulant factors. Considering the above observations, novel molecules to rebalance the hemostatic system in PWH have been developed to be less burdensome and more effective. These novel agents act to rebalance the coagulation system by enhancing coagulation (emicizumab)85or inhibiting anticoagulant pathways (eg, fitusiran, concizumab).86,87
Where are recombinant FVIII concentrates produced?
The first-generation recombinant FVIII concentrates were produced in Chinese Hamster Ovary (CHO) cells, transfected with human cDNA encoding for FVIII in a culture medium containing human albumin.27,28To improve the purity of recombinant FVIll concentrates, albumin was removed from the culture media (second-generation rFVIII concentrates), and finally, albumin was eliminated from the final formulation and a non-protein stabilizer added prior to lyophilization (third-generation rFVIII).29Soon afterward the introduction of the first rFVIII concentrates, rFIX concentrate was also introduced for HB treatment.30At about the same time, a modified rFVIII molecule, in which the B domain had been deleted31or truncated was marketed and showed improved production yield from the cell line and, in the following years, became the standard procedure to produce rFVIII concentrates.32Unfortunately, after the widespread conversion from plasma-derived FVIII (pdFVIII) to rFVIII concentrates for the therapy of HA, the incidence of neutralizing antibodies directed to the clotting activity of FVIII, so-called “inhibitors” became more evident. This adverse event is very severe because the effect of the infused products can be entirely or partially reduced, according to the titer of inhibitor. The incidence of inhibitors is 24% in HA patients treated with pdFVIII concentrates but 33% and up to 52%33–35of those treated with rFVIII concentrates, depending on the study. The high incidence of inhibitors against rFVIII in previously untreated patients (PUPs) was also observed in the regulatory clinical trials for approval of all rFVIII concentrates: the incidence was 29.7% for Kogenate®(Bayer), 31.0% for Recombinate®(Baxter), 33.0% for ReFacto®(Pfizer), and 38.0% after the first 18 exposure days to Kogenate®.36,37The claimed lower immunogenicity of pdFVIII versus rFVIII concentrates was contradicted by a prospective study of PUPs conducted in Germany in which no difference between pdFVIll (36%) and rFVIll (36%) concentrates was observed.38Other retrospective or observational studies showed discordant results between pdFVIII and rFVIII. A randomized, controlled, open-labeled trial in PUPs with severe hemophilia A (Survey of Inhibitors in Plasma-Product Exposed Toddlers [SIPPET]) was conducted in Europe, Asia and the US in which the incidence of inhibitors after the first exposures to pdFVIII or rFVIII concentrates was compared.39The cumulative incidence of FVIII inhibitors observed in 29 HA patients treated with pdFVIII was 26.8% vs 44.5% in 47 HA treated with rFVIII concentrates, respectively. The hazard ratio of developing inhibitors was 1.87 (95% Cl: 1.17–2.96) for rFVIII vs pdFVIII concentrates. Similar outcomes were observed from a study conducted in France.40In conclusion, the immunogenicity of both pdFVIII and rFVIII remains a significant, unsolved adverse event of replacement therapy of HA. After treating HB with pdFIX or rFIX concentrates, FIX inhibitors’ incidence is exceptionally low, about 3%.41These antibodies can fix the complement, and, in some patients, severe anaphylactic reactions occurred just after the intravenous (iv) infusion of the concentrate.42
How often should I give a FVIII?
The prominent feature limiting the benefit of prophylaxis has been the need for frequent, repeated iv infusions every 2–3 days for FVIII and every 5–7 days for FIX concentrates, respectively. The pharmaceutical industry has developed some biochemical methods to increase the HL of concentrates in an effort to reduce the burden of the treatment on patients, especially for young patients. The ability to prolong the plasma half-life and extend the interval between injections followed the application of methods to conjugate the factor molecule with the fragment crystallizable (Fc) of IgG1 or albumin or by adding polyethylene glycol (PEG), which has led to a significant extension of the half-life of concentrates, especially for rFIX. Modifications were first applied to rFIX:
Why is it important to prevent bleeding with replacement therapy?
The aim of replacement therapy is to prevent fatal bleeding and the effects of bleeding on the joints and muscles . Some patients can recognize the symptoms forecasting the bleeding, the so-called “aura.” However, prevention of bleeding is better than treating bleeding with on-demand replacement therapy because, in the short-run, joint inflammation leads to new bleeding and starts a vicious circle that ends in severe arthropathy . Therefore, prevention of bleeding, so-called prophylaxis, was hypothesized to result in better outcomes than on-demand therapy, especially if a minimum trough level was maintained in the plasma. Maintaining the trough level of FVIII or FIX above 3–5 IU/dL is sufficient to reduce the number of bleeding events to one or two episodes every year.48
How to treat bleeding disorders?
Treatment Options for Bleeding Disorders 1 Standard half-life therapies: Standard half-life therapies are used to treat hemophilia A and B, some types of von Willebrand disease, and some rare factor disorders. Dosing can be anywhere from three times a week to every day, depending on the person. 2 Extended half-life (EHL) therapies: EHL contains a molecule that has been modified in some way to delay the breaking down of factor in the body. This results in higher levels of factor in the body lasting for longer, resulting in less frequent infusions. How long the factor is effective in the body depends on the person. Extended half-life therapies are mostly used to treat hemophilia A and B. 3 Bypassing agents are used to treat bleeds in people with hemophilia with inhibitors. These treatments contain other factors that can stimulate the formation of a clot and stop bleeding.
What is extended half life therapy?
Extended half-life therapies are mostly used to treat hemophilia A and B. Bypassing agents are used to treat bleeds in people with hemophilia with inhibitors. These treatments contain other factors that can stimulate the formation of a clot and stop bleeding.
What is EHL therapy?
Extended half-life (EHL) therapies: EHL contains a molecule that has been modified in some way to delay the breaking down of factor in the body. This results in higher levels of factor in the body lasting for longer, resulting in less frequent infusions. How long the factor is effective in the body depends on the person.
Is emicizumab a vasopressin?
Emicizumab was approved by the FDA to treat people with hemophilia A with inhibitors in 2017 and for people with hemophilia A without inhibitors in 2018. Read MASAC's recommendation on emicizumab. Desmopressin (DDAVP) is the synthetic version of vasopressin, a natural antidiuretic hormone that helps stop bleeding.
Home Remedies and Lifestyle
Over-The-Counter (OTC) Therapies
Prescriptions
Specialist-Driven Procedures
Specialist to consult
Complementary and Alternative Medicine
- There is no cure for hemophilia B, but it’s possible for you and your family to learn how to prevent injury and administer your own clotting factor treatments at home. Whether or not you are on home treatment, you should always have replacement clotting factors at home. This is what you will need in case of emergency. After you receive factor treatment, rest, ice, compression, and el…
Summary
- Bleeding into the joints and bruising can be painful. Unfortunately, common OTC pain medications like aspirin and Advil or Motrin IB can interfere with the body’s ability to clot blood. Therefore, you may want to substitute these medications with Tylenol (acetaminophen) for pain relief.2
A Word from Verywell
- While there is no cure for hemophilia B, the disease can be treated over the long term with prescription replacements for the clotting factor IX that is missing in children and adults with this condition. Other medications may also be prescribed to help promote blood clotting. Treatments include: 1. Clotting factor IX products: An estimated 75% of ...