Document Type

Article

Publication Date

6-10-2025

Identifier

DOI: 10.1200/JCO-24-01841; PMCID: PMC12140909

Abstract

PURPOSE: Hematopoietic stem cell transplantation (HSCT) is used as consolidation for pediatric patients with high-risk AML in first complete remission (CR1). The definition of high-risk AML has evolved considerably over the past two decades with the successive identification of new unfavorable risk factors. We conducted a cross-study analysis to determine whether HSCT improves the outcomes of patients with contemporarily defined high-risk AML.

METHODS: We combined data from AAML0531 and AAML1031, the last two phase III clinical trials completed by the Children's Oncology Group (COG). These two trials established the prognostic importance of measurable residual disease (MRD) and several high-risk cryptic cytogenetic/molecular (CM) alterations, which were applied to reclassify patients in the current COG phase III clinical trial, AAML1831. We compared the outcomes after HSCT in CR1 with those after chemotherapy alone in CR1 in the redefined high-risk group.

RESULTS: Our study cohort comprised 463 patients with high-risk CM alterations and 72 patients with standard-risk (SR) CM results with positive MRD at end of induction I. In all, 33.9% and 45.8% of these groups underwent HSCT in CR1, respectively. HSCT was associated with decreased relapse and improved disease-free survival (DFS) in both groups. In the high-risk CM group, 5-year DFS was 26.0% (95% CI, 20.6 to 31.6) and 49.8% (95% CI, 41.7 to 57.4; P < .001) in patients receiving chemotherapy alone and HSCT, respectively. In the SR CM and MRD+ groups, DFS was 16.9% (95% CI, 4.3 to 36.7) compared with 50.9% (95% CI, 32.7 to 66.5; P = .032). HSCT was also associated with improvement in outcomes based on multivariable analysis and across subgroups defined by clinical trial and by high-risk CM subtype, with the exception of chromosome 7 or 5 loss.

CONCLUSION: HSCT was associated with improved outcomes in pediatric patients with contemporarily defined high-risk AML.

Journal Title

Journal of clinical oncology : official journal of the American Society of Clinical Oncology

Volume

43

Issue

17

First Page

1961

Last Page

1971

MeSH Keywords

Humans; Hematopoietic Stem Cell Transplantation; Child; Leukemia, Myeloid, Acute; Male; Female; Child, Preschool; Adolescent; Neoplasm, Residual; Infant; Treatment Outcome; Risk Factors; Disease-Free Survival; Remission Induction

PubMed ID

40294366

Keywords

Hematopoietic Stem Cell Transplantation; Acute Myeloid Leukemia; Residual Neoplasm; Treatment Outcome; Risk Factors; Disease-Free Survival; Remission Induction

Comments

Grants and funding



This article is available under the Creative Commons CC-BY-NC-ND license and permits non-commercial use of the work as published, without adaptation or alteration provided the work is fully attributed.

Publisher's Link: https://doi.org/10.1200/jco-24-01841

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