• DOI:10.1182/blood-2010-10-313908
• Corpus ID: 7086865

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

@article{Bussel2011ARD,
  title={A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia.},
  author={James B Bussel and George R. Buchanan and Diane J Nugent and David J. Gnarra and Lisa R. Bomgaars and Victor S. Blanchette and Yow-Ming C Wang and Kun Nie and Susie Jun},
  journal={Blood},
  year={2011},
  volume={118 1},
  pages={
          28-36
        }
}

• J. Bussel, G. Buchanan, +6 authors S. Jun
• Published 7 July 2011
• Medicine
• Blood

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 × 10(9)/L and 250 × 10(9)/L. A platelet count ≥ 50 × 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 × 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 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.