Combination of triple drug therapy with horse anti-thymocyte globulin, cyclosporine and eltrombopag in severe aplastic anemia: a retrospective observational cohort study.
DOI:
https://doi.org/10.51168/bp99tz87Keywords:
severe aplastic anemia, horse anti-thymocyte globulin, cyclosporine, eltrombopag, immunosuppressive therapy, hematological response, transfusion independenceAbstract
Background
Severe aplastic anemia (SAA) is a life-threatening acquired bone marrow failure disorder characterized by profound pancytopenia. The addition of eltrombopag to horse anti-thymocyte globulin (h-ATG) and cyclosporine may improve hematological recovery.
Aim
To evaluate hematological response, transfusion independence, survival, and treatment-related adverse events associated with triple therapy in patients with SAA.
Methods
This retrospective observational cohort study included 100 patients with SAA treated with h-ATG, cyclosporine, and eltrombopag at SCB Medical College, Cuttack, Odisha, between 2018 and 2026. Hematological response was assessed at 3 and 6 months and categorized as complete, partial, or no response. Transfusion requirements, adverse events, relapse, clonal evolution, and survival were also evaluated.
Results
The mean age was 29.8 ± 14.2 years, with 61% males. Overall hematological response increased from 54% at 3 months to 72% at 6 months, while complete response increased from 14% to 31%. Patients aged ≤40 years had higher response rates than those aged >40 years (78.7% vs. 52.0%, p=0.018). Red-cell and platelet transfusion independence were achieved in 63% and 68% of patients, respectively. The most frequent adverse events were infections (29%), transaminitis (17%), hyperbilirubinemia (14%), and renal dysfunction (11%). Estimated 2-year overall survival was 86%.
Conclusion
Triple therapy was associated with substantial hematological recovery and transfusion independence in patients with SAA.
Recommendation
Prospective multicentre studies with longer follow-up are recommended to validate these findings and assess long-term outcomes
References
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Copyright (c) 2026 Dr. Manamohan Biswal, Dr. Rajeeb Kumar Nayak, Dr. Mitanjali Behera
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