“Eltrombopag Combined with Immunosuppressive Therapy in Pediatric Very Severe Aplastic Anemia: Feasibility and Real-World Outcomes from a Single-Center Study”
摘要
Severe aplastic anemia (SAA) in children is a life-threatening bone marrow failure disorder. While hematopoietic stem cell transplantation (HSCT) offers curative potential, its applicability in low- and middle-income countries (LMICs) is limited by donor availability, cost, and infrastructure. Immunosuppressive therapy (IST) remains the primary treatment for many children. The addition of eltrombopag to standard IST has improved outcomes in adult trials; however, pediatric real-world data from India are limited. We prospectively observed 10 consecutive children (2–18 years) with newly diagnosed severe aplastic anemia treated between January 2022 and June 2024 at a tertiary government hospital in central India. All patients received first-line triple IST comprising horse antithymocyte globulin (hATG), cyclosporine, and eltrombopag. Hematologic response, overall survival (OS), event-free survival (EFS), relapse, and treatment-related toxicities were assessed. At 12 months, overall hematologic response was observed in 5 patients (50%), including complete response in 3 (30%) and partial response in 2 (20%). Two early deaths occurred due to infection-related complications. One-year OS was 80%, and EFS was 50%. Eltrombopag was generally well tolerated; one patient developed grade 1 transaminitis that resolved following dose reduction without treatment interruption. No clonal evolution was observed during follow-up. Triple immunosuppressive therapy with hATG, cyclosporine, and eltrombopag is feasible and associated with encouraging outcomes in pediatric SAA managed in a resource-limited setting. Larger multicenter studies with longer follow-up are required to confirm long-term efficacy and safety.