This Meta-Analysis Evaluates the Optimal Timing for Initiating and Discontinuing Eltrombopag for Aplastic Anemia

Background: Aplastic anemia (AA) is a bone marrow failure syndrome. How to achieve better efficacy of Eltrombopag (EPAG) and IST in the treatment of AA is still unclear. This meta-analysis compares the efficacy of EPAG+IST verses IST alone, and assesses the optimal timing and duration for starting EPAG in different ages of AA patients. Methods: The literature was retrieved from Chinese and English databases up to July 1, 2025. The analysis was conducted using RevMan 5.4 software, employing a fixed effects model to calculate odds ratios (ORs) and 95% confidence intervals (CIs) for the outcomes. The I² statistic was used to assess heterogeneity among included studies. Results: 21 studies involving 2,239 patients were included. The overall response rate in the EPAG+IST group was higher at 3 months (OR = 2.13, 95% CI 1.65–2.74, p < 0.00001) and 6 months (OR = 2.13, 95% CI 1.73–2.61, p < 0.00001). There was no significant difference between two groups at 12 months (OR = 1.14, 95% CI 0.86–1.51, p = 0.36, I² = 35%). For adults, it is recommended the concurrent therapy of EPAG and IST, and continue for a period of 3 months to achieve significant hematological improvement (HI). If EPAG was added after 7 days, patients will achieve HI for about 6 months. For children, regardless of the concurrent or nonconcurrent therapy, it is recommended to continue for about 6 months to achieve better HI. Conclusion: Eltrombopag should be used as early as possible, as it shows a better HI compared with simply IST treatment within 3-6 months, with no significant difference in risk. Concurrent therapy of EPAG should be performed and extending EPAG treatment beyond 12 months may not yield significantly enhanced benefits. Systematic Review Registration: https://www.crd.york.ac.uk/prospero/, identifier CRD42024604778