The treatment and outcome prediction analysis of pediatric acquired severe aplastic anemia.

Abstract

Pediatric acquired severe aplastic anemia (SAA), a prevalent non-malignant hematological disorder, presents significant therapeutic challenges and carries considerable risks. Despite substantial progress in immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (allo-HSCT) in recent years, the protracted treatment duration, substantial costs, and significant disparities in long-term survival outcomes among patients remain problematic. Identifying predictors of treatment response before therapy initiation is crucial for optimal clinical decision-making and complication prevention. Recent studies has pinpointed predictive factors for IST and haploidentical hematopoietic stem cell transplantation (haplo-HSCT) efficacy in SAA, fostering the development and utilization of transplantation-based scoring systems for prognosis evaluation. This review summarizes advancements in treating pediatric SAA and discusses key elements that influence the outcomes of IST and haplo-HSCT, aiming to support clinical decision-making in diverse clinical scenarios.