Introduction: The efficacy of combined cyclosporine (CsA) and antithymocyte/antilymphocyte globulin (ATG/ALG) therapy for aplastic anemia is well-documented; however, its comparative effectiveness against other treatment regimens remains unclear. This systematic review and meta-analysis evaluate the efficacy of combined CsA and ATG/ALG therapy versus alternative treatments in aplastic anemia patients.

Methods: A systematic search was conducted across eight databases (PubMed MEDLINE, Cochrane, Scopus, Web of Science, EMBASE, CINDAHL, CNKI, and Google Scholar) on October 5, 2024. We included randomized controlled trials (RCT's) and cohort studies evaluating CsA plus ATG/ALG efficacy versus alternative regimens in aplastic anemia. Statistical analysis employed random-effects model using R version 3.4.3.

Results: A total of ten studies (n=1,182) were identified, from which selected studies qualified for meta-analysis based on comparator arm compatibility. CsA plus ATG/ALG demonstrated a pooled remission rate of 66% (95% CI: 0.59-0.73; I²=28%) and a five-year overall survival rate of 80% (95% CI: 0.73-0.86; I²=0%). Treatment was associated with a relapse rate and infection rate of 62% (95% CI: 0.54-0.68; I²=0%) and 46% (95% CI: 0.24-0.69; I²=89.3%), respectively. Bleeding and clonal evolution were notably low at 4% (95% CI: 0.02 to 0.08, I² = 0%) and 5% (95% CI: 0.03 to 0.09, I² = 0%), respectively.

Conclusion: Combined CsA and ATG/ALG therapy demonstrated improved survival and remission rates aplastic anemia patients, with minimal bleeding and clonal evolution. The 80% five-year survival rate underscores the long-term benefits of this approach. Future research is required evaluate this combination's superiority against existing regimens and address relapse rate reduction strategies.

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2025
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