Hemophilia is a recessive genetic disease of X chromosome. Most of the patients are men, and about 80% of them are hemophilia A, which lacks the eighth coagulation factor. The eighth coagulation factor is a kind of protein which can coagulate blood. Because of the lack of coagulation factors, it is easier to bleed than ordinary people and needs long-term preventive treatment. Patients may experience repeated severe bleeding at joints, which may eventually lead to joint injury, other diseases and other problems, which will have an impact on patients' physiology and psychology. The most common are social activities or fear and worry about sports.
Bispecific monoclonal antibody drugs can reduce the bleeding attack of hemophilia A.
Dr. Wang Jiande, director of the Center for Rare Diseases and Hemophilia in Taichung Veterans General Hospital, explained that the US Food and Drug Administration (FDA) recently quickly passed a "bispecific monoclonal antibody" preventive drug for hemophilia A patients, which was used to prevent or reduce the number of bleeding episodes in adults and children with positive antibodies to coagulation factor VIII. It is a great medical progress for doctors who treat hemophilia and a very good opportunity for patients' health care.
Director Wang Jiande further explained that in the course of treatment, some hemophilia A patients will produce "Factor VIII antibody", and this immune antibody reaction will affect the effectiveness of the currently available therapeutic drugs and make the treatment in trouble. Although there are therapeutic drugs in use at present, the effect is still far from that of patients who do not produce antibodies, which will also cause a lot of inconvenience.
Clinical trial: In subjects over 12 years old, the number of bleeding can be reduced by 87%.
Among subjects younger than 12 years old, 87% patients did not experience bleeding.
The new drug is a "bispecific monoclonal antibody" developed by patented antibody engineering technology. Its main function is to simulate the function of the eighth coagulation factor, which can bridge other coagulation factors in the blood to restore the coagulation function in the blood, and treat hemophilia A. Clinical trials show that compared with subjects who did not use preventive treatment, subjects who received preventive treatment with new drugs can reduce the number of bleeding by 87%, from an average of 23.3 bleeding per year to only 2.9 bleeding per year. In another clinical trial for people under the age of 12, 87% of the subjects did not have bleeding events under preventive treatment. At the same time, the quality of life of patients was analyzed. After using this new drug, patients can not only improve symptoms related to hemophilia, such as pain, swelling and joint pain, but also improve physical functions, such as exercise pain and walking difficulties.
Prevention of new therapy subcutaneous injection to improve patient convenience
Director Wang Jiande said that with the development of medicine, we have a lot of expectations for the new treatment of hemophilia A. Compared with the previous treatment, the number of injections has been greatly reduced, from bleeding treatment several times a week to preventive injection once a week. In addition, all the treatments in the past were intravenous injection, and the new preventive treatment was changed to subcutaneous injection, which greatly improved the convenience and compliance of patients. With better prevention and treatment, these patients can participate in social activities and sports that they were worried about before or could not participate in in the future.
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