Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Therapy-Related AML / Myelodysplastic Syndromes
Only a few reports describe the outcome of children undergoing HSCT for t-AML. One study described outcomes of 27 children with t-AML who received related and unrelated donor HSCT. Three-year OS rates were 18.5% ± 7.5% and event-free survival rates were 18.7% ± 7.5%. Poor survival was mainly the result of very high transplant-related mortality (59.6% ± 8.4%). Another study reported a second retrospective single-center experience of 14 patients transplanted for t-AML/t-MDS between 1975 and 2007. Survival was 29%, but in this review only 63% of patients diagnosed with t-AML/t-MDS underwent HSCT. A multicenter study (CCG-2891) looked at outcomes of 24 children with t-AML/t-MDS compared with other children enrolled on the study with de novo AML (n = 898) or MDS (n = 62). Children with t-AML/t-MDS were older and low-risk cytogenetics rarely occurred. Although rates of achieving CR and OS at 3 years were worse in the t-AML/t-MDS group (CR, 50% vs. 72%; P = .016; OS, 26% vs. 47%; P = .007), survival was similar (OS, 45% vs. 53%; P = .87) if patients achieved a CR. The importance of remission to survival in these patients is further illustrated by another single-center report of 21 children undergoing HSCT for t-AML/t-MDS between 1994 and 2009. Of the 21 children, 12 had t-AML (11 in CR at the time of transplant), seven had refractory anemia (for whom induction was not done), and two had refractory anemia with excess blasts. Survival of the entire cohort was 61%; those in remission or with refractory anemia had a disease-free survival of 66%, and for the three patients with more than 5% blasts at the time of HSCT, survival was 0% (P = .015). Because t-AML is rare in children, it is not known whether the significant decrease in transplant-related mortality after unrelated donor HSCT noted over the past several years will translate to improved survival in this population. Patients should be carefully assessed for pre-HSCT morbidities caused by earlier therapies and approaches should be adapted to give adequate intensity while minimizing transplant-related mortality.
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