Transplantation for children with acute myeloid leukemia: a comparison of outcomes with reduced intensity and myeloablative regimens.

Publication Type:

Journal Article


Blood (2014)


2014, Clinical Research Division, February 2014


The safety and efficacy of reduced-intensity conditioning (RIC) regimens for the treatment of pediatric acute myeloid leukemia is unknown. We compared the outcome of allogeneic hematopoietic cell transplantation in children with acute myeloid leukemia using RIC regimens to those receiving myeloablative-conditioning (MAC) regimens. One hundred and eighty patients were evaluated (39 with RIC and 141 with MAC regimens). Results of univariate and multivariate analysis showed no significant differences in the rates of acute and chronic graft versus host disease (GVHD), leukemia-free and overall survival between treatment groups. The 5-year probabilities of overall survival with RIC and MAC regimens were 45% and 48%, respectively (p=0.99). Moreover, relapse rates were not higher with RIC compared to MAC regimens (39% vs. 39%, p=0.95), and recipients of MAC regimens were not at higher risk for transplant-related mortality compared to recipients of RIC regimens (16% vs. 16%, p=0.73). In this relatively modest study population and after carefully controlled analyses, the data support a role for RIC regimens for acute myeloid leukemia in children undergoing allogeneic hematopoietic cell transplantation. The data also provide justification for a designing a carefully controlled randomized clinical trial that examines the efficacy of regimen intensity in this population.