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A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia.

Author(s): Bussel JB, Buchanan GR, Nugent DJ, Gnarra DJ, Bomgaars LR, Blanchette VS, Wang YM, Nie K, Jun S

Affiliation(s): Department of Pediatrics, Division of Hematology, Weill Medical College of Cornell University, New York, NY, USA. jbussel@med.cornell.edu

Publication date & source: 2011-07-07, Blood., 118(1):28-36. Epub 2011 Apr 18.

Publication type: Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't

Romiplostim, a thrombopoietin-mimetic peptibody, increases and maintains platelet counts in adults with immune thrombocytopenia (ITP). In this first study of a thrombopoietic agent in children, patients with ITP of >/= 6 months' duration were stratified by age 1:2:2 (12 months-< 3 years; 3-< 12 years; 12-< 18 years). Children received subcutaneous injections of romiplostim (n = 17) or placebo (n = 5) weekly for 12 weeks, with dose adjustments to maintain platelet counts between 50 x 10(9)/L and 250 x 10(9)/L. A platelet count >/= 50 x 10(9)/L for 2 consecutive weeks was achieved by 15/17 (88%) patients in the romiplostim group and no patients in the placebo group (P = .0008). Platelet counts >/= 50 x 10(9)/L were maintained for a median of 7 (range, 0-11) weeks in romiplostim patients and 0 (0-0) weeks in placebo patients (P = .0019). The median weekly dose of romiplostim at 12 weeks was 5 mug/kg. Fourteen responders received romiplostim for 4 additional weeks for assessment of pharmacokinetics. No patients discontinued the study. There were no treatment-related, serious adverse events. The most commonly reported adverse events in children, as in adults, were headache and epistaxis. In this short-term study, romiplostim increased platelet counts in 88% of children with ITP and was well-tolerated and apparently safe. The trial was registered with http://www.clinicaltrials.gov as NCT00515203.

Page last updated: 2011-12-09

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