HSCT

The BFM group examined outcomes of children with AML over a 35-year period and found that the greatest improvement in overall outcome was the improvement in survival after relapse.[34]

The BFM group examined outcomes of children with AML over a 35-year period and found that the greatest improvement in overall outcome was the improvement in survival after relapse.[34]

Improved EFS after relapse or refractory disease was only seen in patients who received an HSCT as part of their salvage therapy.

Unrelated-donor HSCT has been reported to result in the following:[35][Level of evidence C1]

Unrelated-donor HSCT has been reported to result in the following:[35][Level of evidence C1]

The 5-year probabilities of leukemia-free survival (LFS) were 45%, 20%, and 12% for patients with AML who underwent transplants in second CR, overt relapse, and primary induction failure, respectively.

A number of studies, including a large, prospective Center for International Blood and Marrow Transplant Research (CIBMTR) cohort study of children and adults with myeloid diseases, have shown similar or superior survival with busulfan-based regimens compared with total-body irradiation (TBI) for transplant, including children with a history of CNS-positive disease.[36-40]

Matched sibling-donor transplant has generally led to the best outcomes, but use of single-antigen mismatched related or matched unrelated donors results in very similar survival at the cost of increased rates of GVHD and nonrelapse mortality.[41] Outcomes for patients who received umbilical cord transplants are similar to those in patients who received other unrelated donor transplants. Matching patients at a minimum of 7/8 alleles (HLA A, B, C, DRB1) leads to less nonrelapse mortality.[42] Haploidentical approaches are being used with increasing frequency and have resulted in comparable outcomes to other stem cell sources in pediatrics.[43] Direct comparison of haploidentical and other unrelated donor sources has not been performed in pediatrics, but studies in adults have shown similar outcomes.[44]

Reduced-intensity approaches have been used successfully in pediatrics, but mainly in children unable to undergo myeloablative approaches.[45] A randomized trial in adults showed superior outcomes with myeloablative approaches compared with reduced-intensity regimens.[46]