Romiplostim effective treatment for immune thrombocytopenia

January 01, 0001

Romiplostim effective treatment for immune thrombocytopenia

Romiplostim, a thrombopoietin mimetic, increases platelet counts in patients with immune thrombocytopenia, with few adverse effects. These investigators from multiple countries conducted a manufacturer supported unblended trial of romiplostin vs. standard care for 52-weeks. They randomly assigned 234 adult patients with immune thrombocytopenia, who had not undergone splenectomy, to receive the standard of care (77 patients) or weekly subcutaneous injections of romiplostim (157 patients). Primary end points were incidences of treatment failure and splenectomy. Secondary end points included the rate of a platelet response, safety outcomes, and the quality of life.

They found: "The rate of a platelet response in the romiplostim group was 2.3 times that in the standard-of-care group. Patients receiving romiplostim had a significantly lower incidence of treatment failure (18 of 157 patients = 11%) than those receiving the standard of care (23 of 77 patients = 30%) (odds ratio with romiplostim, 0.31). Splenectomy also was performed less frequently in patients receiving romiplostim (14 of 157 patients = 9%) than in those receiving the standard of care (28 of 77 patients = 36%) (odds ratio, 0.17). The romiplostim group had a lower rate of bleeding events, fewer blood transfusions, and greater improvements in the quality of life than the standard-of-care group. Serious adverse events occurred in 23% of patients (35 of 154) receiving romiplostim and 37% of patients (28 of 75) receiving the standard of care."

The authors concluded: "Patients treated with romiplostim had a higher rate of a platelet response, lower incidence of treatment failure and splenectomy, less bleeding and fewer blood transfusions, and a higher quality of life than patients treated with the standard of care."

This drug is likely to become the standard treatment for immune thrombocytopenia if affordable.

For the full abstract, click here.

N Engl J Med 363:1889-1899, 11 November 2010
© 2010 to the Massachusetts Medical Society
Romiplostim or Standard of Care in Patients with Immune Thrombocytopenia. David J. Kuter, Mathias Rummel, Ralph Boccia, et al. Correspondence to Dr. Kuter: [email protected]

Category: B. Blood/Blood Forming Organs/Immune Mechanisms. Keywords: immune thrombocytopenia, ITP, romiplostim, randomized controlled trial, journal watch.
Synopsis edited by Dr Linda French, Toledo, Ohio. Posted on Global Family Doctor 23 November 2009

Pearls are an independent product of the Cochrane primary care group and are meant for educational use and not to guide clinical care.