December 9, 2009

Nplate Evaluated in Children with Chronic ITP


Among children with chronic immune thrombocytopenic purpura (ITP; an autoimmune disorder characterized by low platelet counts), treatment with Nplate® (romiplostim) stimulates platelet production and appears to be well tolerated. These results were presented at the 2009 annual meeting of the American Society of Hematology.

Platelets are cells that help blood clot. Thrombocytopenia occurs when platelet levels fall below normal. It can often occur as a direct result of cancer, from cancer treatment, or from other diseases such as ITP. ITP is an autoimmune disorder characterized by low levels of platelets. The condition leaves patients at risk of serious bleeding events.

Nplate is a drug designed to increase platelet production. The U.S. Food and Drug Administration (FDA) has approved Nplate for the treatment of thrombocytopenia in adult patients with chronic ITP that does not respond to standard therapies.

To evaluate Nplate for the treatment of pediatric ITP, researchers conducted a Phase I/II study among 22 children with chronic ITP. Seventeen of the children were treated with Nplate, and five were treated with placebo.

Two efficacy endpoints were assessed: 1) a platelet count of over 50,000 platelets per microliter for two consecutive weeks during the treatment period; and 2) a platelet count of 20,000 platelets per microliter above baseline for two consecutive weeks.

  • Nplate was generally well tolerated; rates of adverse events were similar among children treated with Nplate and children treated with placebo.
  • 88% of the children treated with Nplate achieved both efficacy endpoints during treatment. None of the children treated with placebo achieved either efficacy endpoint.

These results are promising and suggest that Nplate may eventually play a role in the treatment of chronic ITP in children.

Reference: Buchanan GR, Bomgaars L, Bussel JB et al. A Randomized, Double-Blind, Placebo-Controlled Phase 1/2 Study to Determine the Safety and Efficacy of Romiplostim in Children with Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP). Presented at the 51st annual meeting of the American Society of Hematology. December 5-8, 2009. New Orleans, LA. Abstract 680.

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