title: A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia.
abstract: 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.
authors.affiliation: Department of Pediatrics, Division of Hematology, Weill Medical College of Cornell University, New York, NY, USA. jbussel@med.cornell.edu
authors.forename: James B
authors.initials: JB
authors.lastname: Bussel
authors.forename: George R
authors.initials: GR
authors.lastname: Buchanan
authors.forename: Diane J
authors.initials: DJ
authors.lastname: Nugent
authors.forename: David J
authors.initials: DJ
authors.lastname: Gnarra
authors.forename: Lisa R
authors.initials: LR
authors.lastname: Bomgaars
authors.forename: Victor S
authors.initials: VS
authors.lastname: Blanchette
authors.forename: Yow-Ming
authors.initials: YM
authors.lastname: Wang
authors.forename: Kun
authors.initials: K
authors.lastname: Nie
authors.forename: Susie
authors.initials: S
authors.lastname: Jun
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D007223:Infant; D007279:Injections, Subcutaneous; D008297:Male; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D011961:Receptors, Fc / Q000008:administration & dosage*; D053628:Receptors, Thrombopoietin / Q000819:agonists*; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics; D034061:Thrombopoiesis / Q000187:drug effects*; D013926:Thrombopoietin / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics; D016896:Treatment Outcome
chemical_list: D011961:Receptors, Fc; D053628:Receptors, Thrombopoietin; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim