During the gene therapy trial , 13 people with hemophilia A from across the U. Test results show that over 1 year after receiving their treatment, all 13 patients have been able to stop their previous treatment, and 11 are showing normal or near-normal levels of clotting factor.
In a linked editorial , Dr. Marijke van den Berg, from the Julius Centre for Health Sciences and Primary Care at University Hospital Utrecht in the Netherlands, discusses the significance of this gene therapy trial, and also of another with hemophilia B patients. One of the things that needs clarifying, explains Dr.
Also, because the trial excluded people whose immune systems may reject the virus used to carry the corrective gene the AAV vector , who had no history of developing factor VIII inhibition, and who were free of hepatitis , it is not clear whether the treatment would work for most people with hemophilia A.
Another important implication that Dr. It is so exciting. Read this article in Spanish. If blood does not clot, life-threatening bleeding can occcur. Find out about how blood clots, how clotting factors work, and how treatment has….
A look at hemarthrosis, a condition where there is bleeding around the joint cavity. Here we learn more about its symptoms, treatment and causes. Who is at Risk for Hemolytic Anemia? How is Hemolytic Anemia Diagnosed? How is Hemolytic Anemia Treated? What Are the Signs and Symptoms of Hemophilia? How is Hemophilia Diagnosed? How is Hemophilia Treated? Who is at Risk for Immune Thrombocytopenia? Who is at Risk of Iron-Deficiency Anemia? What are the Symptoms of Iron Deficiency?
How is Iron-Deficiency Anemia Diagnosed? How is Iron-Deficiency Anemia Treated? What Causes Pernicious Anemia? What are the Risk Factors for Pernicious Anemia? What Causes Polycythemia Vera? How is Polycythemia Vera Treated? How is Pulmonary Embolism Treated? What Causes Sickle Cell Disease? Who is at Risk for Sickle Cell Disease? How Is Hemophilia Treated? Treatment With Replacement Therapy The main treatment for hemophilia is called replacement therapy.
Complications of Replacement 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 Antibodies to the clotting factor. Researchers are studying new ways to deal with antibodies to clotting factors. However, the risk of getting an infectious disease from human clotting factors is very small due to: Careful screening of blood donors Testing of donated blood products Treating donated blood products with a detergent and heat to destroy viruses Vaccinating people who have hemophilia for hepatitis A and B Damage to joints, muscles, and other parts of the body.
Delays in treatment can cause damage such as: Bleeding into a joint. If this happens many times, it can lead to changes in the shape of the joint and impair the joint's function. Swelling of the membrane around a joint. Pain, swelling, and redness of a joint. Pressure on a joint from swelling, which can destroy the joint. Home Treatment With Replacement Therapy You can do both preventive ongoing and demand as-needed replacement therapy at home. Home treatment has several advantages: You or your child can get quicker treatment when bleeding happens.
Early treatment lowers the risk of complications. Fewer visits to the doctor 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. Antifibrinolytic Medicines Antifibrinolytic medicines including tranexamic acid and epsilon aminocaproic acid may be used with replacement therapy.
Gene Therapy Researchers are trying to find ways to correct the faulty genes that cause hemophilia. Treatment of a Specific Bleeding Site Pain medicines, steroids, and physical therapy may be used to reduce pain and swelling in an affected joint. Which Treatment Is Best for You? Mild hemophilia—Replacement therapy usually isn't needed for mild hemophilia. Moderate hemophilia—You may need replacement therapy only when bleeding occurs or to prevent bleeding that could occur when doing certain activities.
Back to Haemophilia. Most cases of haemophilia are severe and need preventative treatment. This involves regular injections of clotting factor medicine. If your child has haemophilia, you'll be trained to give them the injections when they're young. They'll be taught how to inject themselves when they're older, to help avoid regular hospital appointments. In some cases, injections may be given into a device called an implantable port, which can be surgically placed under the skin.
This port is connected to a blood vessel near the heart, so you do not need to try to find a vein for every injection. People who have preventative treatment will need regular follow-up appointments with their care team so their progress can be monitored.
Preventative treatment is usually continued for life. It may be possible for someone to change to on-demand treatment, but they may be advised to switch back to preventative treatment if they have any episodes of significant bleeding.
There are different types of haemophilia. This page covers the most common types: haemophilia A and haemophilia B. They have similar symptoms but need different treatments because different clotting factors are affected.
Preventative treatment for haemophilia A involves regular injections of a medicine called octocog alfa Advate. Read about octocog alfa Advate on the European Medicines Agency's website. This medicine is an engineered version of clotting factor VIII 8 , the clotting factor people with haemophilia A do not have enough of.
0コメント