A prospective multi-center study of 243 children with high-risk AML in CR1 undergoing allogeneic HCT (n=141) or autologous HCT (n=102), depending on availability of an HLA-compatible sibling donor. Infants, patients with AML-M7, or complex karyotype or those with FLT3-ITD, were also eligible to be transplanted from alternative donors: umbilical cord blood (n=26) or matched unrelated donor (n=75). All patients received a myeloablative regimen combining busulfan, cyclophosphamide and melphalan. The study found comparable 8-year disease-free survival (DFS) in allogeneic and autologous transplantation: 73% vs. 63%, respectively (p=n.s.). The cumulative incidence of transplant-related mortality was 7% in both groups. In 26 patients transplanted with umbilical cord blood, the 8-year DFS was 92.3%. The authors conclude that both autologous and allogeneic HCT, after a preparative regimen of busulfan, cyclophosphamide, and melphalan, offer a chance of cure for a large proportion of children with AML in CR1.
Autologous, Allogeneic HCT Both Effective in Pediatric High-Risk AML