New Drug Promising for Immune Thrombocytopenia
Few adverse side effects seen in phase 1-2 trial of the new thrombopoietic agent, AMG 531
THURSDAY, Oct. 19 (HealthDay News) -- The thrombopoiesis-stimulating agent, AMG 531, appears to be safe and effective for boosting platelet counts in patients with immune thrombocytopenic purpura, according to a preliminary study in the Oct. 19 issue of the New England Journal of Medicine.
James Bussel, M.D., of Weill Medical College of Cornell University in New York City and colleagues conducted a phase 1-2 study to determine the safety and efficacy of AMG 531 during and after treatment of immune thrombocytopenia patients with continued low platelet counts after primary therapy. While most treatments for immune thrombocytopenia are aimed at reducing platelet destruction, AMG 531 is designed to boost megakaryocyte production of platelets.
No major adverse effects were seen during the treatment of 41 patients that could be definitely attributed to AMG 531. Four patients had transient post-treatment worsening of thrombocytopenia. Platelet counts returned to baseline after treatment was discontinued. Ten of 16 patients receiving low doses of AMG 531 in the phase 2 study (1 or 3 micrograms per kilogram of body weight per week for six weeks) achieved the targeted range of platelet counts.
"Thrombopoietic agents represent a promising new therapeutic strategy for immune thrombocytopenia that is refractory to second- and third-line therapies," wrote Michael Bromberg, M.D., Ph.D., in an accompanying editorial. "These agents might also serve as an alternative for patients who cannot tolerate immunosuppressive therapy or who are not candidates for it."
The study was funded by Amgen, the manufacturer of AMG 531.