In a Children’s Oncology Group (COG) study (AAML1831) reported in Journal of Clinical Oncology, Huang et al found that hematopoietic stem cell transplantation (HSCT) was associated with improved outcomes in pediatric patients with high-risk acute myeloid leukemia (AML).
Study Details
The study involved combining data from the two prior phase III trials in AML (AAML0531 and AAML1031), which demonstrated the prognostic importance of measurable residual disease (MRD) and several high-risk cryptic cytogenetic/molecular (CM) alterations in defining high-risk disease. These findings were used to reclassify high risk in the current trial. Outcomes after HSCT in first complete remission (CR1) were compared with those after chemotherapy alone in CR1 in the redefined high-risk group and the standard-risk group.
Key Findings
The study cohort consisted of 463 patients with high-risk CM alterations and 72 patients with standard-risk CM results and positive MRD at end of induction I.
A total of 33.9% of patients with high-risk disease and 45.8% of those with standard-risk disease underwent HSCT in CR1. In the high-risk CM groups, 5-year disease-free survival was 49.8% (95% confidence interval [CI] = 41.7%–57.4%) among patients receiving HSCT vs 26.0% (95% CI = 20.6%–31.6%) among those receiving chemotherapy alone (P < .001).
In the standard-risk CM and MRD-positive groups, 5-year disease-free survival was 50.9% (95% CI = 32.7%–66.5%) among patients receiving HSCT vs 16.9% (95% CI = 4.3%–36.7%) among those receiving chemotherapy alone (P = .032).
HSCT was associated with improved outcomes across subgroups and by high-risk CM subtype, with the exception of chromosome 7 or 5 loss.
The investigators concluded: “HSCT was associated with improved outcomes in pediatric patients with contemporarily defined high-risk AML.”
Benjamin J. Huang, MD, of the Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco, is the corresponding author for the Journal of Clinical Oncology article.
Disclosure: The study was supported by the National Cancer Institute, St Baldrick’s Foundation Consortium Grant, and others. For full disclosures of the study authors, visit ascopubs.org.
