Factors influencing the treatment response of cyclosporine-a therapy in aplastic anemia patients in resource-limited settings

Abstract

Background and aim: Cyclosporine-A (CsA) is the preferred treatment for Aplastic Anemia (AA) in developing countries with restricted access to Antithymocyte Globulin (ATG) and Matched Sibling Donor Hematopoietic Stem Cell Transplantation (MSD HSCT). Treatment response to CsA is variable, and this study aims to determine the factors influencing the treatment response of CsA in AA patients. Methods: A cross-sectional analytical observational study included 40 AA patients diagnosed according to Camitta criteria, who had CsA therapy for 3 months at the Hematology-Oncology Outpatient Clinic at Wahidin-Sudirohusodo Hospital, Indonesia, from June 2023 to June 2025. The treatment response was evaluated using the British Society for Haematology’s 2024 Guideline. The examined factors comprised age, body mass index (BMI), diagnosis-to-treatment interval, number of blood transfusions preceding therapy, corticosteroid administration, comorbidities (hypertension and diabetes mellitus), and disease severity. Multivariate logistic regression analysis was used to identify variables influencing CsA treatment response. Results: This study reported a 40% response rate (RR) to CsA therapy (27.5% partial response and 12.5% complete response). A significant correlation was identified between the number of blood transfusions preceding therapy (P = 0.022; OR = 7.328; CI 1.341-40.056) and disease severity (P = 0.040; OR = 12.081; CI 1.122-130.054) concerning CsA treatment response within 3 months. Conclusions: Our findings suggest that AA patients who received ≥3 blood transfusions preceding therapy and exhibited severe disease were less likely to respond to CsA therapy. This highlights the prognostic importance of transfusion history and disease severity in predicting treatment outcomes.

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