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

James B. Bussel, George R. Buchanan, Diane J. Nugent, David J. Gnarra, Lisa R. Bomgaars, Victor S. Blanchette, Yow Ming Wang, Kun Nie, Susie Jun

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137 Scopus citations


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 × 109/L and 250 × 10 9/L. A platelet count ≥ 50 × 109/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 × 109/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 μg/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.

Original languageEnglish (US)
Pages (from-to)28-36
Number of pages9
Issue number1
Publication statusPublished - Jul 7 2011


ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

Bussel, J. B., Buchanan, G. R., Nugent, D. J., Gnarra, D. J., Bomgaars, L. R., Blanchette, V. S., ... Jun, S. (2011). A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia. Blood, 118(1), 28-36. https://doi.org/10.1182/blood-2010-10-313908