Allogeneic hematopoietic stem cell transplantation is associated with improved long-term survival and molecular MRD clearance in KMT2A-altered AML.

Authors

Yi Chen, Zhiwei Ding, Shuxia Zhang, Xiaofan Li, Ruyu Cai, Danhui Fu, Fenge Yang, Shaoyuan Wang, Yong Wu, Nainong Li, Jing Zheng

Published in

Annals of medicine. Volume 58. Issue 1. Pages 2758994. Epub Nov 12, 2026.

Abstract

KMT2A-altered acute myeloid leukemia (AML) is a rare, aggressive high-risk subtype. The prognostic value of flow-cytometric phenotypic MRD versus KMT2A gene-specific molecular MRD, and the role of allogeneic hematopoietic stem cell transplantation (Allo-HSCT), remain poorly defined.
This single-center retrospective cohort enrolled 155 newly diagnosed adolescent and adult patients with KMT2A-altered AML. Treatment response, MRD status, and survival were analyzed by Kaplan-Meier and multivariate Cox regression; time-dependent Cox regression was used as a sensitivity analysis to account for guarantee-time bias.
Among 153 evaluable patients, the complete remission rate was 70.7% and overall response rate 78.9%, with median RFS of 12.4 months and OS of 16.4 months. After excluding transplanted patients, the three non-transplant regimens yielded similar survival, indicating no inherent advantage of induction regimens. Flow-MRD-negative patients had superior OS, and concurrent KMT2A molecular MRD negativity conferred the best outcomes. Allo-HSCT was associated with a markedly higher rate of sustained molecular MRD negativity (74.6% vs. 14.4%, p < 0.0001). On multivariate analysis, persistent molecular MRD positivity and absence of Allo-HSCT independently predicted inferior RFS and OS; after time-dependent adjustment, Allo-HSCT showed only a non-significant trend, while molecular MRD positivity remained the dominant adverse prognostic factor.
In KMT2A-altered AML, sustained KMT2A molecular MRD negativity is the key determinant of favorable long-term survival. Allo-HSCT promotes molecular MRD clearance and may serve as a consolidation strategy to achieve deep molecular remission.

PMID:
43016283
Bibliographic data and abstract were imported from PubMed on 12 Nov 2026.

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