Efficacy of combination immunotherapy of eltrombopag, ATG and cyclosporine in aplastic anaemia

Abstract Introduction: Aplastic anemia (AA) is a rare, life-threatening bone marrow failure disorder with annual prevalence of 0.5 to 6 cases per million people globally, with higher incidence in Asia. It is characterized by pancytopenia and hypocellular marrow. While immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporine has been a standard of care for ineligible patients of hematopoietic stem cell transplantation, the addition of Eltrombopag, a thrombopoietin receptor agonist, is a promising drug to enhance the treatment response. Objective: To evaluate the efficacy and safety of combination immunotherapy with Eltrombopag, ATG, and Cyclosporine in patients with AA. Methodology: The study assess the recent prospective and retrospective studies including randomized controlled trials and cohort analyses assessing triple therapy efficacy. The study focuses on response rates (complete and partial), time to hematologic recovery, relapse rates, and adverse effects. Results: The addition of Eltrombopag to standard IST significantly improved overall hematologic response. The studies have shown 75% overall response rate at 6 months with complete response rate of 46%. The studies indicated median time to first response of 8.1 weeks, and relapse rates remained low at 15% over 12–24 months follow-up. No significant cytogenetic abnormalities noted in short- to mid-term follow-up. Conclusion: Triple immunotherapy with Eltrombopag, ATG, and Cyclosporine shows a promising and well-tolerated first-line treatment for severe AA as it has faster, deeper, and more sustained hematologic responses with minimal side effects. These findings support the incorporation of Eltrombopag into frontline management for patients with AA. Large scale clinical trials are required to assess durability of response and late clonal risks.