Efficacy and Safety of Romiplostim in the Treatment of Aplastic Anemia: A Systematic Review and Single-Arm...

Romiplostim, a second‑generation thrombopoietin receptor agonist (TPO‑RA), has been increasingly investigated for aplastic anemia (AA) across various populations and treatment settings; however, its overall efficacy, optimal dose, safety profile, and sources of heterogeneity across studies have not been systematically quantified and validated. This study aims to perform an up‑to‑date systematic review and single‑arm meta‑analysis to comprehensively assess the overall efficacy and safety of romiplostim in AA patients.

We systematically searched PubMed, Embase, Web of Science, Cochrane Library, and CNKI from inception to March 3, 2026, without language restrictions. Two independent reviewers screened literature, extracted data, and assessed methodological quality using the Newcastle-Ottawa Scale (NOS) and Methodological Index for Non-Randomized Studies (MINORS). A random-effects model was applied in Stata 15.1 to pool overall response rate (ORR) and complete response rate (CRR); subgroup, sensitivity, and publication bias analyses were performed to explore heterogeneity and robustness.

Nineteen studies with 382 patients were included. The pooled ORR was 72.4% (95% CI: 61.9%-81.9%), and CRR was 25.5% (95% CI: 18.4%-33.1%). Patients receiving 20 μg/kg romiplostim achieved higher ORR (77.6% vs. 51.7%) and CRR (29.3% vs. 17.0%) than those receiving 10 μg/kg (P = 0.081). Patients aged <60 years showed higher ORR (74.9% vs. 49.3%) than those ≥60 years (P = 0.298). Tolerability was favorable, with low discontinuation rate (2.1%) and no treatment-related deaths.

Romiplostim demonstrates promising efficacy and safety in AA, with enhanced outcomes at 20 μg/kg and in younger patients. Further high-quality trials are warranted.