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Romiplostim in children with chronic refractory ITP: randomized placebo controlled study
Mohsen Saleh Elalfy1, Abeer Ahmed Abdelmaksoud, Khadiga Yehia Eltonbary
1Pediatric Hemostasis-Thrombosis, Ain Shams University, Cairo, Egypt. elalfym@hotmail.com
Insights
Romiplostim effectively increased platelet counts in children with chronic idiopathic thrombocytopenic purpura (ITP). The treatment was safe and well-tolerated, showing significant efficacy in refractory cases.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacology
Background:
- Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disorder characterized by low platelet counts.
- Chronic ITP in children often requires effective and safe treatment options, especially for refractory cases.
- Romiplostim, a thrombopoietin receptor agonist, stimulates platelet production.
Purpose of the Study:
- To evaluate the safety and efficacy of romiplostim in pediatric patients with chronic ITP.
- To assess romiplostim's effectiveness in children refractory to other treatments.
Main Methods:
- A randomized, placebo-controlled trial involving 18 children with chronic ITP.
- Patients received romiplostim or placebo for 12 weeks, with dose adjustments.
- Efficacy endpoint defined as platelet count > 50,000/µL.
Main Results:
- 83.3% of patients on romiplostim achieved the efficacy endpoint.
- Adverse events were similar between romiplostim and placebo groups (50%), with no serious events.
- Romiplostim demonstrated good tolerability and efficiency in treating chronic refractory ITP.
Conclusions:
- Romiplostim is a safe and effective treatment for children with chronic refractory ITP.
- The drug facilitates sustained platelet count increases in pediatric patients.
- Romiplostim offers a viable therapeutic option for a challenging pediatric hematological condition.
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.
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