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Treatment of Hemophilia
Treatment of Hemophilia
Hemophilia is a rare bleeding disease that is characterized by a shortage of clotting factors that are responsible for forming the blood clots to stop bleeding. Replacement therapy is the primary treatment for hemophilia that supplies the clotting factors to patients with hemophilia. It can help to combat a bleeding episode that is in progress. It can also be administered on a regular basis to prevent bleeding episodes at home.The clotting factor treatments for replacement therapy can be either derived from human blood or synthetically generated in a laboratory.
Recombinant Factor Concentrate
Recombinant clotting factors are synthetically generated factors in the laboratory and they resemble healthy humans clotting factors. They are considered the best choice for treatment.
Recombinant factors VIII including Kovaltry, Kogenate, Advate, Afstyla, Nuwig, Recombinant, and Xyntha are used for treating hemophilia A. Recombinant factors IX including BeneFix, Ixinity, and Rixubis are used for treating hemophilia B.
These factors can be modified to make them more stable and live longer. These longer-living molecules help the patients to reduce the frequency of injections. PEGylation is one of the ways to increase the stability of the recombinant clotting factors by attaching the molecule polyethylene glycol (PEG) to the clotting factor. The PEGylated clotting factors approved for the treatment of hemophilia A are Bax 855, Adynovate, and N8-GP. PEGylated clotting factor approved for the treatment of hemophilia B is Refixia (N9-GP).
Plasma-derived Factor Concentrates
The liquid part of blood containing proteins like albumin, antibodies, and clotting factors is called Plasma. Clotting factors can be purified from the plasma of healthy donors for the treatment of hemophilia patients. Hemofil-M is used for the treatment of hemophilia A and AlphaNine SD and Mononine are used for treating hemophilia B.
Hemophilia can be treated with a prescription hormone named desmopressin. It resembles vasopressin, a naturally occurring hormone that stimulates the release of clotting factor VIII. Formulations of desmopressin like Stimate and DDAVP are used for the treatment of mild hemophilia A.
An antibody called Hemlibra binds clotting factors IXa and X. Combining these two factors molecules helps in facilitating clotting.
People with hemophilia who are treated with clotting factors may develop inhibitors against these factors. This results in making the treatment ineffective. So, Bypassing agents are used in these patients as an alternative treatment. Even obizur (recombinant factor VIII) derived from pigs is used to treat this disorder. Both Feiba that helps to convert prothrombin into thrombin that is involved in the formation of blood clots and NovoSeven RT that resembles human clotting factor VIIa responsible for promoting the production of fibrin, a protein required for clot formation is used for the treatment of several forms of hemophilia.