Magazine article Drug Topics

Scientists at ASH Unveil New Tools to Manage Bleeding and Clotting

Magazine article Drug Topics

Scientists at ASH Unveil New Tools to Manage Bleeding and Clotting

Article excerpt

Researchers at the American Society of Hematology (ASH) annual meeting evaluated new approaches to managing major bleeding and clotting diseases. These complications affect millions of people worldwide. Here are highlights from the meeting, held recently in Adanta, that created substantial buzz:

Amgen Inc. scientists reported data from a randomized, pivotal Phase III study showing that their novel peptibody AMG 531 (romiplostim) boosts and sustains platelet counts in splenectomized adult patients with chronic immune thrombocytopenic purpura (ITP)-an autoimmune disorder characterized by low blood platelet numbers, bruising, and bleeding. It affects about 200,000 Americans and a similar number of Europeans.

Also found was that some patients receiving romiplostim together with standard treatments (e.g., corticosteroids) could stop taking the latter.

The pioneering study is the first to show that ITP is at once a disease of increased platelet destruction and suboptimal platelet production-not platelet destruction alone as scientists had previously believed, according to lead investigator Terry Gernsheimer, M.D., associate professor of medicine, division of hematology, University of Washington School of Medicine, Seattle.

"Patients with chronic ITP produce antiplatelet autoantibodies that destroy their blood platelets and, at times, also damage their megakaryocytes (cells that produce platelets in the bone marrow)," she explained. "Splenectomy, high-dose steroids, androgens, and newer immunosuppressive drugs treat refractory ITP - but not always successfully. Some experts say splenectomy succeeds in 60% of cases; others say, much less often....

"Romiplostin is an investigational therapy that stimulates platelet production at the thrombopoietin receptor," continued Gernsheimer. "Thrombopoietin is a hormone that encourages the bone marrow to produce platelets."

Amgen recently filed for regulatory approval of romiplostin to treat adult thrombocytopenia in the United States, European Union (EU), Australia, Canada, and Japan. The drug has orphan designation in all those areas, Gernsheimer pointed out.

In her team's pivotal 63-patient Phase III study reported in a plenary session at ASH, either romiplostin or placebo was injected weekly for 24 weeks at a starting dost of 1mcg/kg, and adjusted to maintain a target platelet count based on weekly response. …

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