New Breakthrough for Hemophilia Patients

News Health Articles - New Breakthrough for Hemophilia Patients. People with hemophilia have blood that is difficult to freeze due to gene abnormalities. In severe cases, people with hemophilia can experience spontaneous internal bleeding, even in the brain. Now there are new methods for patients with hemophilia blood clotting.

Researchers used gene therapy and successfully treat six patients with blood clotting disorders, namely severe hemophilia. The study is still preliminary and involve only 6 patients.



Single infusion using a new treatment given to some patients for more than a year, can improve their blood clotting ability significantly.

"The study is remarkable progress," said Dr.. Ronald Crystal, chairman of genetic medicine at New York City's Weill Cornell Medical College as reported from MSNHealth, Tuesday (13/12/2011).

"The study is still a landmark study," said Dr.. Katherine Ponder, from Washington University School of Medicine physician.

He praised the study in an editorial accompanying the publication of research in the New England Journal of Medicine. The research has also been presented at the conference of the American Society of Hematology in San Diego.

Hemophilia is a genetic disease which is a blood clotting disorder that potentially life-threatening. Hemophilia most often occurs in men. If bleeding occurs in the joints, it can cause movement problems weakness and pain.

Trial of gene therapy in hemophilia patients, showed an increased blood clotting time, but only a few weeks.

"We can not make blood clots in people with hemophilia is longer than just a few weeks," said Val Bias, chief executive of the National Hemophilia Foundation.

Experts say the new method should be tested on more patients to confirm whether these methods proved effective and there is no risk attached to them. Even if all goes well, these methods are still a few more years to be available to patients with hemophilia.

Since the late 1960s, doctors have given intravenously in patients with hemophilia with clotting proteins. The method of treatment has been quite successful, which can increase the lifetime of hemophilia patients an average of up to 63 years.

But for severe cases, treatment may involve 2 or 3 infusions per week and cost more than $ 250,000 per year.

The new study led by researchers from University College London Cancer Institute and St. Jude Children's Research Hospital in Memphis, Tennessee. All the study participants, amounting to 6 is a man from London who has a severe form of hemophilia type B. Each study participant received a healthy infusion of genetic material for 20 minutes, which is derived from viruses found in monkeys.

Viruses are often used to transport DNA into cells. Increasing the amount of clotting proteins in the blood of study participants is less than 1 percent of normal levels or at least 2 percent, and in one case as much as 11 percent.

That number may seem very little, but enough to help patients with hemophilia, and 4 research participants can stop conventional treatment altogether.

However, it is not clear about the risks of such treatment methods. In one patient, liver enzyme levels increased up to 5 times the normal levels.

It does not cause symptoms, but there are concerns about liver inflammation and the patient has been treated with steroids. The researchers noted it may be able to trigger the treatment of hepatitis in some patients.