Advancements in Hemophilia Treatment

Information on the Advances in Hemophilia Treatment

Studies have shown that hemophilia is a rare genetic bleeding disorder that most commonly occurs in males. The bleeding often goes into a person’s joints and muscles. There is Hemophilia A, which accounts for up to 84 percent of individuals who have hemophilia. This is a result of a defect in a person’s gene coding that affects coagulation. There is also Hemophilia B. This is often referred to as Christmas disease. It is caused by a defect in the genes responsible for the synthesis of coagulation. These defects minimize a person’s ability to produce the protein essential for clotting. This is essential for a person’s body to be able to stop bleeding.

Advancements in Hemophilia Treatment


In the United States, approximately 1 in 5,000 male children will have hemophilia. It is estimated that as many as 400 people are born with it annually. Around 33 percent of those born with hemophilia do not have a genetic link to the disease. According to recent research, only one person with hemophilia out of seven is 45 years old or older.

Hemophilia Treatment

There have been significant advancements in the treatment of hemophilia during the past few decades. At one time, hemophilia was considered a fatal disease. It is now a disorder with an identifiable molecular basis. This makes it possible to create safe and effective treatments. It remains a lifelong bleeding disorder, but genetic therapy trials and more research are moving closer towards a cure.

Prophylaxis Treatment

This is given to individuals with hemophilia and are drugs that act as a replacement clotting factor. They are used in anticipation of a situation where bleeding may happen. This treatment first started in 1992 and has been improved since then. This treatment is able to help people with severe hemophilia improve their condition to mild. This requires it to be given to patients three times a week. It is done even if they don’t have a bleeding episode.

Gene Therapy

There have also been promising advances in the use of gene therapy for treating hemophilia. In a large percentage of patients, this treatment has been able to significantly decrease their spontaneous bleeding. This decrease has been able to be maintained in most cases for over three years. It has been successful with many patients who have a severe hemophilia condition. When gene therapy has been used, it has resulted in a 90 percent decrease in bleeding episodes. It has also caused the reduction or elimination of the use of prophylaxis


There has been success in the development of blood products that contain no human or animal products. This is a medication that can be administered using a capsule. It has led to a high level of improvement with plasma-derived products. This has been very successful when dealing with all of the safety concerns associated with hemophilia.


Compliance with treatment has also increased. This is attributed to the successful education of the parent and patient about hemophilia. More people are beginning to comprehend the short and long term impact of following treatment requirements. Studies have shown that over 58 percent of parents treat their child’s hemophilia as required by a treatment plan. It also found that more than 38 percent of parents did not properly follow their child’s treatment plan. Improved awareness of the early signs of bleeding is important. Knowing the long-term consequences from untreated bleeds is essential. Computer-based education is helping improve patient awareness. Instructional programs and other aids are being provided. Summer camps for children with hemophilia are increasing. Support groups for parents to help learn about treatments and stay organized are also becoming more common.