The genomic landscape of relapsed infant and childhood KMT2A-rearranged acute leukemia

L Louise Ahlgren M Mattias Pilheden H Helena Sturesson G Guangchun Song (Key Laboratory of Agro-products Quality and Safety Control in Storage and Transport Process, Ministry of Agriculture and Rural Affairs) M Michael P. Walsh M Minjun Yang M Maud Maillard H Huanbin Zhao Z Zhongshan Cheng V Varsha Singh A Anders Castor C Cornelis Jan Pronk H Hanne Vibeke Marquart B Birgitte Lausen P Pauline Schneider G Gisela Barbany K Katja Pokrovskaja Tamm J Jonas Abrahamsson O Olli Lohi L Linda Fogelstrand P Pablo Menendez R Rob Pieters J Jinghui Zhang K Karin Lindkvist-Petersson J Jun J. Yang (Department of Pharmacy and Pharmaceutical Sciences) T Tanja A. Gruber R Ronald W. Stam J Jing Ma (State Key Laboratory of Coordination Chemistry, School of Chemistry) A Anna K. Hagström-Andersson

Abstract

Abstract To study the mechanisms of relapse in KMT2A-rearranged (KMT2A-r) acute lymphoblastic (ALL) and acute myeloid leukemia (AML), we performed whole-genome and exome sequencing of infants and children with relapsed ALL/AML (n = 36), and longitudinal deep-sequencing of 257 samples in 30 patients. Somatic alterations in drug-response genes, most commonly in TP53 and IKZF1 (64%), were highly enriched in early relapse ALL (79%, 9-36 months after diagnosis), but rare in very early relapse ALL (<9 months, 9%). A marked chemotherapy-exposure signature was detected for mutations in early relapse ALL but not in very early ALL or AML relapse, in line with different mechanisms of relapse. Longitudinal analyses could track residual leukemia cells, clonal drug responses, and the upcoming relapse. These results highlight that KMT2A-r ALL and AML evade therapy differently and provide insights into the mechanisms of relapse in this highly lethal form of pediatric acute leukemia.

Article Details

Volume / Issue Vol. 16, Issue 1
Published October 08, 2025
ISSN 2041-1723
Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (29)

L

Louise Ahlgren

M

Mattias Pilheden

H

Helena Sturesson

G

Guangchun Song

Key Laboratory of Agro-products Quality and Safety Control in Storage and Transport Process, Ministry of Agriculture and Rural Affairs

M

Michael P. Walsh

M

Minjun Yang

M

Maud Maillard

H

Huanbin Zhao

Z

Zhongshan Cheng

V

Varsha Singh

A

Anders Castor

C

Cornelis Jan Pronk

H

Hanne Vibeke Marquart

B

Birgitte Lausen

P

Pauline Schneider

G

Gisela Barbany

K

Katja Pokrovskaja Tamm

J

Jonas Abrahamsson

O

Olli Lohi

L

Linda Fogelstrand

P

Pablo Menendez

R

Rob Pieters

J

Jinghui Zhang

K

Karin Lindkvist-Petersson

J

Jun J. Yang

Department of Pharmacy and Pharmaceutical Sciences

T

Tanja A. Gruber

R

Ronald W. Stam

J

Jing Ma

State Key Laboratory of Coordination Chemistry, School of Chemistry

A

Anna K. Hagström-Andersson