title: Romiplostim in children with chronic refractory ITP: randomized placebo controlled study.
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 × 10(9)/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.
authors.affiliation: Pediatric Hemostasis-Thrombosis, Ain Shams University, Cairo, Egypt. elalfym@hotmail.com
authors.forename: Mohsen Saleh
authors.initials: MS
authors.lastname: Elalfy
authors.forename: Abeer Ahmed
authors.initials: AA
authors.lastname: Abdelmaksoud
authors.forename: Khadiga Yehia
authors.initials: KY
authors.lastname: Eltonbary
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D006801:Humans; D008297:Male; D010919:Placebos; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D011961:Receptors, Fc / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use*; D016037:Single-Blind Method; D013926:Thrombopoietin / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D010919:Placebos; D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim