Evaluation of Safety and Efficacy of Romiplostim in the Treatment of Pediatric Immune Thrombocytopenia: A Real-World Observational Study

Abstract

Romiplostim, a thrombopoietin receptor agonist, is a second-line therapy for primary immune thrombocytopenia (ITP). This real-world observational study evaluated romiplostim safety and efficacy in 52 children with persistent/chronic ITP. Baseline and follow-up data were retrospectively collected from patients' files. Primary endpoints: platelet response rate and time to first response. Secondary endpoints: durable response rate, safety, and health-related quality of life (HRQoL) using (PedsQL 4.0). Romiplostim decreased significant bleeding frequency (from 76.9% to 34.6%, P=0.003), hospital admission rate (from 69.2% to 7.7%, P<0.001), and rescue medication use (from 65.4% to 11.5%, P<0.001). In all, 92.3% of patients achieved platelet response in a median of 1 month, and 80.8% achieved a durable response. The most frequent adverse events were headache and joint pain; no serious adverse events occurred. Platelet count was positively correlated with HRQoL in parent-proxy reports (P<0.05). In linear regression univariate analysis, platelet count ≥100×109/L was associated with better parent-proxy HRQoL (P=0.004) but not after multivariate adjustment, while joint pain remained independently associated with lower parent-proxy HRQoL. Beyond confirming the efficacy and safety of romiplostim, this study supports the sequential use of thrombopoietin receptor agonists, as romiplostim was still effective in patients who did not respond to previous treatment with eltrombopag.