Hematopoietic stem cell transplantation in pediatric acute leukemia: Current applications, challenges, and future directions

Hematopoietic stem cell transplantation (HSCT) has become a cornerstone in the treatment of pediatric acute leukemia, particularly for those with high-risk or relapsed disease. This review provides a comprehensive analysis of HSCT in pediatric acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), tracing its historical development from its inception in the mid-20th century to its current status as a potentially curative therapy. It details the clinical applications of HSCT, including specific indications for transplantation based on risk stratification and minimal residual disease monitoring. The paper also addresses the critical challenge of post-transplantation relapse—a leading cause of treatment failure—exploring strategies such as optimized conditioning regimens, graft manipulation, and post-transplant maintenance therapies to improve outcomes. Furthermore, we examine the future of HSCT, highlighting innovations in donor selection, immunotherapy (e.g., CAR-T cell therapy), and gene editing that promise to enhance efficacy and accessibility. In general, by synthesizing current evidence and emerging trends, this review aims to inform clinical decision-making and stimulate further research to optimize HSCT for children with acute leukemia.