The genomic landscape of relapsed infant and childhood KMT2A-rearranged acute leukemia
(2025) In Nature Communications 16(1).- 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,... (More)
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.
(Less)
- author
- organization
- publishing date
- 2025-10-08
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- Humans, Myeloid-Lymphoid Leukemia Protein/genetics, Histone-Lysine N-Methyltransferase/genetics, Leukemia, Myeloid, Acute/genetics, Infant, Precursor Cell Lymphoblastic Leukemia-Lymphoma/genetics, Child, Preschool, Child, Female, Male, Gene Rearrangement, Recurrence, Mutation, Exome Sequencing, Ikaros Transcription Factor/genetics, Tumor Suppressor Protein p53/genetics, Genomics, Adolescent
- in
- Nature Communications
- volume
- 16
- issue
- 1
- article number
- 8964
- pages
- 14 pages
- publisher
- Nature Publishing Group
- external identifiers
-
- pmid:41062506
- scopus:105018250401
- ISSN
- 2041-1723
- DOI
- 10.1038/s41467-025-64190-8
- language
- English
- LU publication?
- yes
- additional info
- © 2025. The Author(s).
- id
- 5b19b447-6d2f-4a92-8e2f-921adf3b71da
- date added to LUP
- 2025-10-20 11:32:09
- date last changed
- 2025-12-02 07:11:32
@article{5b19b447-6d2f-4a92-8e2f-921adf3b71da,
abstract = {{<p>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.</p>}},
author = {{Ahlgren, Louise and Pilheden, Mattias and Sturesson, Helena and Song, Guangchun and Walsh, Michael P and Yang, Minjun and Maillard, Maud and Zhao, Huanbin and Cheng, Zhongshan and Singh, Varsha and Castor, Anders and Pronk, Cornelis Jan and Marquart, Hanne Vibeke and Lausen, Birgitte and Schneider, Pauline and Barbany, Gisela and Pokrovskaja Tamm, Katja and Abrahamsson, Jonas and Lohi, Olli and Fogelstrand, Linda and Menendez, Pablo and Pieters, Rob and Zhang, Jinghui and Lindkvist-Petersson, Karin and Yang, Jun J and Gruber, Tanja A and Stam, Ronald W and Ma, Jing and Hagström-Andersson, Anna K}},
issn = {{2041-1723}},
keywords = {{Humans; Myeloid-Lymphoid Leukemia Protein/genetics; Histone-Lysine N-Methyltransferase/genetics; Leukemia, Myeloid, Acute/genetics; Infant; Precursor Cell Lymphoblastic Leukemia-Lymphoma/genetics; Child, Preschool; Child; Female; Male; Gene Rearrangement; Recurrence; Mutation; Exome Sequencing; Ikaros Transcription Factor/genetics; Tumor Suppressor Protein p53/genetics; Genomics; Adolescent}},
language = {{eng}},
month = {{10}},
number = {{1}},
publisher = {{Nature Publishing Group}},
series = {{Nature Communications}},
title = {{The genomic landscape of relapsed infant and childhood KMT2A-rearranged acute leukemia}},
url = {{http://dx.doi.org/10.1038/s41467-025-64190-8}},
doi = {{10.1038/s41467-025-64190-8}},
volume = {{16}},
year = {{2025}},
}
