Allogeneic stem cell transplantation for children with acute myeloid leukemia in second complete remission

Area of Science:

• Pediatric Hematology Oncology
• Stem Cell Transplantation
• Acute Myeloid Leukemia Research

Background:

• Relapsed acute myeloid leukemia (AML) in children presents a significant therapeutic challenge.
• Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potential curative option for refractory AML.
• Evaluating outcomes of unmanipulated allo-HSCT in pediatric AML is crucial for treatment refinement.

Purpose of the Study:

• To assess the efficacy and safety of unmanipulated allo-HSCT in children with relapsed AML.
• To identify factors influencing overall survival, leukemia-free survival, relapse, and transplant-related mortality.
• To determine the impact of donor type and graft-versus-host disease on allo-HSCT outcomes in pediatric AML.

Main Methods:

• Retrospective, multicenter analysis of 63 children with relapsed AML receiving allo-HSCT in second complete remission.
• Data collection on donor type (matched family vs. unrelated), stem cell source, and preparative regimens.
• Multivariate analysis to identify predictors of survival, relapse, and transplant-related mortality.

Main Results:

• Five-year overall survival and leukemia-free survival were 53% and 49%, respectively.
• Cumulative incidence of relapse and transplant-related mortality were 26% and 25%, respectively.
• Matched family donors predicted better leukemia-free survival; chronic graft-versus-host disease and older age at diagnosis increased transplant-related mortality.

Conclusions:

• Unmanipulated allo-HSCT can rescue a significant proportion of children with relapsed AML, particularly with matched family donors.
• Leukemia recurrence and transplant-related mortality remain key contributors to treatment failure.
• Optimizing donor selection and graft-versus-host disease management strategies are essential for improving allo-HSCT outcomes, especially with unrelated donors.