Molecular landscape and risk stratification in acute myeloid leukemia – insights from the real-world REFORM-AML cohort
Year: 2026
Type: preprint
Study design: Observational Study
Abstract: ABSTRACT Large genomically annotated acute myeloid leukaemia (AML) datasets exist, but population-based contemporary cohorts remain scarce. Here we report clinicopathological, genomic, and outcome data from Danish AML patients. 2,512 AML patients were identified between 2015-2022, of whom 33.8% had available NGS data (NGS+). In patients ≤70 years, baseline characteristics and outcomes were comparable between NGS+ and NGS− groups. In patients >70 years, more NGS+ patients received intensive treatment, but survival was similar among intensively treated patients. The distribution of mutations varied significantly by age and sex, with older age and male sex exhibiting higher frequencies of adverse-risk gene mutations. In intensively treated NGS+ patients, ELN2017 stratified 5-year OS: 58.4% (favorable), 43.4% (intermediate), and 28.2% (adverse), with hazard ratios (HRs) of 0.63 (favorable) and 1.45 (adverse) relative to intermediate. ELN2022 yielded corresponding OS rates of 56.9%, 51.8%, and 29.7%, with HRs of 0.78 and 1.86. The two models had comparable predictive performance for OS in a time-dependent model. In conclusion, outcomes of intensively treated AML patients were comparable irrespective of NGS status, underscoring the representativeness of the REFORM-AML database for the Danish AML population. Age and male sex correlated with adverse-risk mutations, and both ELN2017 and ELN2022 robustly predicted survival.
Source: medRxiv
Authors: Daniel Tuyet Kristensen, Rasmus Froberg Brøndum, Michael Knudsen, Lykke Grubach, Claus Marcher, Birgitte Preiss, Maria Lyng Bibi, Estrid Høgdall, Tim Svenstrup Poulsen, Vibe Skov, Andreas Due Ørskov, Kirsten Grønbæk, Jakob Werner Hansen, Claudia Schöllkopf, Jack Bernard Cowland, Mette Klarskov, Marianne Tang Severinsen, Christopher Vejgaard, Ole Halfdan Larsen, Søren Vang, Martin Bøgsted, Anne Stidsholt Roug
Institutions: Aarhus University, Aalborg University Hospital, Aarhus University Hospital, Aalborg University, University of Southern Denmark, Odense University Hospital, Herlev Hospital, Copenhagen University Hospital, Roskilde Sygehus, Zealand University Hospital, University of Copenhagen, Rigshospitalet
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W7204937703