12/12/2011

SAN DIEGO, CA – Patients with a common congenital bleeding disorder known as von Willebrand Disease (VWD) who are unresponsive or allergic to the preferred treatment may respond well to modest doses of an anti-inflammatory drug, according to a study led by Margaret Ragni, M.D., Ph.D., professor of medicine and clinical translational research with the University of Pittsburgh School of Medicine. The results will be presented at the 53rd annual meeting of the American Society of Hematology (ASH) on Dec. 12.

VWD is caused by a deficiency of von Willebrand factor (VWF) in the blood. This factor helps blood platelets clump together and stick to the blood vessel walls. Affecting men and women equally, VWD often is mild in nature and may cause nosebleeds, heavy periods or postoperative bleeding. Aspirin and nonsteroidal anti-inflammatory drugs can worsen the condition.

“While the majority of patients with VWD respond well to the preferred treatment, Desmopressin, approximately 20 percent have adverse reactions,” said Dr. Ragni. “For this patient population, treatment options are quite limited and involve a significant time commitment. We wanted to see if this subset of patients might respond to recombinant interleukin-11 (rhIl-11). We ultimately found that daily, low-dose injections could be self-administered and were safe and effective in patients with VWD, as well as type A hemophilia.”

The phase II study enrolled healthy patients with either VWD or mild hemophilia A in non-bleeding states. Patients received 25 microgram/kg of rhIl-11 daily for four days, which increased blood levels of VWF, as well as factor VIII levels, an indicator that the drug could be useful in bleeding situations.

“This drug appears to be a potential alternative treatment option, and it looks like rhIl-11 might not only serve as a safe alternative, but could also save patients significant time and inconvenience when compared to the other options,” said Dr. Ragni.

She will conduct further rhIl-11 studies, including a phase three study for women with heavy periods, and hopes the trials will solidify rhIl-11 as a viable treatment alternative for people with certain bleeding disorders.

The study was sponsored by the University of Pittsburgh Vascular Medicine Institute.