Annals of Hematology

, Volume 90, Issue 11, pp 1341–1344

Romiplostim in children with chronic refractory ITP: randomized placebo controlled study

  • Mohsen Saleh Elalfy
  • Abeer Ahmed Abdelmaksoud
  • Khadiga Yehia Eltonbary
Original Article

DOI: 10.1007/s00277-011-1172-9

Cite this article as:
Elalfy, M.S., Abdelmaksoud, A.A. & Eltonbary, K.Y. Ann Hematol (2011) 90: 1341. doi:10.1007/s00277-011-1172-9

Abstract

Romiplostim stimulates thrombopoietin receptor to increase platelet production of megakaryocytes in idiopathic thrombocytopenic purpura (ITP). This study aimed to evaluate the safety and efficacy of romiplostim in children with chronic ITP. Eighteen patients with chronic ITP, either none responsive or failed to maintain response on two or more therapeutic modalities, were enrolled. Patients were randomized (2:1) to receive romiplostim or placebo for 12 weeks, initiated at 1 μg/kg/week, escalated to 5 μg/kg/week, and then tapered. Median patients' age was 8.5 years, and the median baseline platelet count (PC) was 10.5 × 109/L. The median weekly dose of romiplostim was 2 μg/kg. Fifty percent of patients in both romiplostim and placebo arms had at least one adverse event (AE); none was serious. Ten patients on romiplostim (83.3%) maintained the efficacy endpoint (PC > 50,000). Romiplostim was well-tolerated and efficient in treating the children with chronic refractory ITP with no unexpected AEs.

Keywords

Chronic ITPPediatricsRomiplostim

Abbreviations

ITP

Immune thrombocytopenia

PC

Platelet count

AE

Adverse event

Copyright information

© Springer-Verlag 2011

Authors and Affiliations

  • Mohsen Saleh Elalfy
    • 1
    • 2
  • Abeer Ahmed Abdelmaksoud
    • 1
  • Khadiga Yehia Eltonbary
    • 1
  1. 1.Pediatric Hemostasis-ThrombosisAin Shams UniversityCairoEgypt
  2. 2.Department of Pediatric HematologyAin Shams University CairoCairoEgypt