Initiation timing and treatment duration of eltrombopag added to immunosuppressive therapy in aplastic anemia: a systematic review and meta-analysis.

BACKGROUND: Aplastic anemia (AA) is a bone marrow failure syndrome. Whether the combination of TPO-RAs and IST is superior to IST alone is an ongoing debate. This meta-analysis compares the efficacy of EPAG+IST versus IST alone, and assesses the initiation timing and treatment duration of eltrombopag (EPAG) across different age groups of AA patients. METHODS: The literature was retrieved from Chinese and English databases up to October 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 the two groups at 12 months (OR = 1.14, 95% CI 0.86 - 1.51, P = 0.36, I² = 35%). CONCLUSION: The addition of EPAG to IST may improve early hematological responses at 3 and 6 months in patients with AA, with no significant difference at 12 months. Concurrent initiation (within 7 days) was associated with earlier responses in adults. SYSTEMATIC REVIEW REGISTRATION: https://www.crd.york.ac.uk/prospero/, identifier CRD42024604778.