Background

Aplastic anemia (AA) is a bone marrow failure syndrome. Whether the combination of TPO-RAs and IST is superior to IST alone is ongoing debate. This meta-analysis compares the efficacy of Eltrombopag (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 May 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 subgroup analysis, 1) For adults, it is recommended the concurrent therapy of EPAG and IST (EPAG was added within 7 days of the start of IST treatment), and continue for a period of 3 months to achieve significant HI. If EPAG was added after 7 days, patients will achieve HI for about 6 months. 2) For children, regardless of the concurrent or nonconcurrent therapy, there was no significant difference compared with IST alone in 3 months. It is recommended to continue for about 6 months to achieve better HI. 3) For all AA patients, the HIs of additional Eltrombopag for more than 12 months were not significantly different from those of simply IST.

Conclusion

Our study indicates that Eltrombopag should be used as early as possible, as it shows a better hematological improvements (HIs) 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

KEY WORDS

Aplastic anemia (AA), Bone marrow failure (BMF), Eltrombopag(EPAG), Immunosuppressive therapy (IST), Thrombopoietin receptor agonists (TPO-RA)

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2025
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