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.

Immune Thrombocytopenia - Current Diagnostics and Therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI

“…There is no standard of care. TRAs are effective in chronic ITP in children and adolescents [49,52,84,[131][132][133]. Eltrombopag has been approved for this indication since 2016.…”

Immune thrombocytopenia in children and adults: what's the same, what's different?

“…12 In the light of these data, the first published studies with thrombomimetics in children with chronic ITP should be considered with caution in order to avoid underestimating the risk of early reticulin deposition in the bone marrow. 13,14 A substantial fraction of patients with ITP undergo spontaneous remission within three to six months after the initial diagnosis. These patients have formerly been referred to as "acute" while those with persistent low counts are referred to as "chronic".…”

Romiplostim in children with chronic immune thrombocytopenia (ITP): the French Experience

“…In a short randomized trial, Bussel et al (2011) reported efficacy in the treated group (17 patients) versus placebo (five patients), with 88% of patients reaching a platelet count >50 9 10 9 /l for 2 weeks in the romiplostim group versus 0% in the placebo group. Such encouraging results were confirmed in another study where romiplostim was administrated to 12 patients for 12 weeks (Elalfy et al, 2011). In a recent paediatric study, seven patients with nonresponsive chronic ITP were treated by romiplostim, of whom four had a R and only one a durable one (Mokhtar et al, 2012).…”