Romiplostim significantly boosts platelet counts and reduces rescue therapy in immune thrombocytopenia.

Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia and an elevated risk of bleeding. While initial first-line therapies, such as corticosteroids and intravenous immunoglobulin, offer some benefit, a substantial number of patients either relapse or develop refractory disease. Romiplostim, a thrombopoietin receptor agonist, has emerged as a promising second-line therapeutic option. However, comprehensive evidence directly comparing its efficacy and safety against placebo across both pediatric and adult populations has remained limited, highlighting a critical gap in current treatment guidelines and patient management strategies.

A systematic review and meta-analysis of randomized controlled trials (RCTs) was rigorously conducted following PRISMA guidelines. The search encompassed major databases including PubMed, Scopus, Cochrane Library, and Google Scholar, up to March 2026. The analysis included 9 RCTs with a combined total of n=748 ITP patients, comparing romiplostim against a placebo control. A random-effects model was employed to calculate mean differences (MD) and risk ratios (RR) with 95% confidence intervals (CI) for primary endpoints such as platelet counts and response rates. The Cochrane RoB 2 tool was used to assess the risk of bias in the included studies.