A Complete List of Approved Treatments for Hemophilia

A Complete List of Approved Treatments for Hemophilia

Hemophilia is a rare blood disease that is usually inherited and occurs mostly in men. When you injure yourself, the proteins in your body, known as clotting factors, work together with the platelets to heal the wound. In hemophilia, people have low levels of Factor VIII or Factor IX and hence cannot heal themselves. This can lead to severe internal bleeding. Treatments for hemophilia include replacement therapy, desmopressin, antibodies, and bypassing agents.

Replacement therapy through recombinant clotting factors

One of the best ways to treat hemophilia is to replace the missing factors through human blood or synthetically produced blood in a laboratory. The latter are called recombinant clotting factors and are widely popular as genetically engineered factors that are less likely to transmit infections. For hemophilia A treatment, recombinant factor VIII like Kovaltry, Advate, Xyntha, Kogenate, Afstyla, Nuwig, and Recombinant are used. BeneFix, Ixinity, and Rixubis are used to treat hemophilia B.

Replacement therapy with plasma-derived clotting factors

Missing factors derived through plasma present in the human blood are called plasma-derived clotting factors. Plasma contains proteins like albumin, antibodies, and clotting factors that are purified from donors and used to treat hemophilia patients. Plasma-derived factor VIII treatments include Hemofil-M, Alphanate, HumateP, and Wilate. Products containing factor IX include AlphaNineSD, Mononine, and Hemoleven (only in Europe).

Desmopressin

This is similar to vasopressin, which is a naturally born hormone that enables the growth of clotting factor VIII. This treatment is highly efficient for people who have mild to moderate hemophilia. Two medications known are used for this procedure. DDAVP is given in tablet form or directly injected in the vein, and Stimate is a nasal spray. Once the factor VIII levels up, the patient may not require to use clotting factors to prevent internal bleeding.

Antibodies

A medication known as Hemlibra (also called ACE 910 or emicizumab) works as a direct replacement for factor VIII clotting factor. Given directly under the skin as an injection, Hemlibra is effective in reducing internal bleeding in hemophilia A patients. However, many people develop inhibitors against recombinant factor VIII, making the whole procedure a failure. This medication is most effective with people diagnosed with hemophilia A with inhibitors.

Bypassing agents

The bypassing agents procedure is used as an alternative treatment for those who develop inhibitors. This method also works with patients who have an autoimmune condition called acquired hemophilia. Feiba medication converts prothrombin into thrombin, while NovoSeven RT is similar to human clotting factor VIIa which helps in the production of clot formation. Obizur is derived from pigs and used to treat acquired hemophilia A patients.

Amicar and Cryoprecipitate

Amicar medication helps in preventing blood clots from disintegrating and also results in firmer clots. It is given in pill or liquid form through injection or prescribed orally. Cryoprecipitate is derived from thawing fresh plasma and is rich in factor VIII. However, this treatment is discontinued in our country and many European countries due to safety concerns such as the transmission of infections.