CDC Foundation Announces $3 Million Grant to Fund Hemophilia Study

The CDC Foundation has received a $3 million grant from Wyeth Pharmaceuticals to study why some hemophilia patients do not respond to the drugs currently used to stop or prevent a bleeding episode. The three-year grant will provide funding for scientists at the Centers for Disease Control and Prevention (CDC) to study the risk factors associated with this serious problem.

Hemophilia is an inherited bleeding disorder that results from deficiencies in blood clotting factors. Without treatment, it can lead to spontaneous internal bleeding and excessive bleeding following injuries or surgery. These bleeding episodes can cause severe damage to joints, neurological systems and organ systems involved in a hemorrhage, and, in rare cases, death.

Approximately seven percent of people with hemophilia develop an inhibitor or antibody to the anti-hemophilic drugs that are used to treat an episode. Inhibitors render the drugs used to control the bleeding ineffective.

“Little is known about how or why certain individuals develop an inhibitor,” says J. Michael Soucie, Ph.D., acting associate director for science in CDC’s Division of Hereditary Blood Disorders. “We do know that people with hemophilia inhibitors are twice as likely to be hospitalized for a bleeding complication as hemophilia patients without the inhibitor. The cost of hospital care for a bleeding complication is an average of 10 times greater for those with inhibitors.”

In this study, scientists in CDC’s Division of Hereditary Blood Disorders will collaborate with selected hemophilia treatment centers to gather data about the development of inhibitors and evaluate the risk factors associated with the occurrence. CDC scientists believe that factors such as age, race/ethnicity, type of hemophilia gene defect, frequency of treatment, and the type of drug used for treatment, may play a role in the development of inhibitors. “Understanding the risk factors for inhibitors will help to guide treatment of persons at high risk and may lead to the development of products that are less likely to cause an inhibitor,” adds Dr. Soucie.

About 70 percent of all people with hemophilia in the United States receive at least some of their medical care from federally funded treatment centers located throughout the country. These centers serve more than 24,000 persons with bleeding disorders and offer a range of comprehensive services. CDC scientists work closely with these centers to monitor the safety of the nation’s blood supply for persons being treated with blood products, as well as to monitor the occurrence of joint complications experienced by persons with hemophilia. The new study will tie into this monitoring system and additional blood specimens and data will be collected to study inhibitors.”

CDC scientists have long wanted to determine how and why certain hemophilia patients develop this inhibitor, but have not had the funds for a comprehensive study,” says Charles Stokes, president and CEO of the CDC Foundation. “With the help of Wyeth Pharmaceuticals, CDC can now take the first steps toward ultimately finding a solution to this problem.”

“Current information demonstrates that inhibitors occur with all hemophilia products. However, most of this information is derived from clinical studies which use different definitions and classifications for inhibitors. The availability of a large body of data using consistent classification will be very valuable, and will help to shape future treatment decisions,” says Dr. Gary L. Stiles, executive vice president and chief medical officer of Wyeth Pharmaceuticals.