British journal of haematology
October 18, 2026 ยท via Europe PMC
Subtype- and functional class-dependent prognostic impact of CSF3R mutations in acute myeloid leukaemia: A cohort study of 2500 patients.
An J, Hui Y, Li Y, +14 more, Wei H.
AI summary โ for doctors, generated from the abstract only
CSF3R mutations in AML carry context-dependent prognosis, with the clearest adverse impact in CEBPA-bZip AML.
What was studied
Cohort of 2500 patients with newly diagnosed de novo non-APL AML given intensive induction, comparing outcomes by CSF3R mutation status and functional class.
Key findings
- CSF3R mutations occurred in 3.5% and co-occurred often with CEBPA-bZip mutations and RUNX1::RUNX1T1
- Overall they were linked to inferior relapse-free survival but not OS or EFS
- In CEBPA-bZip AML, EFS HR was 2.76 (p = 0.001) and RFS HR 2.66 (p = 0.002); membrane-proximal mutations had RFS HR 2.27 overall
Clinical relevance
Could refine risk assessment, especially for CEBPA-bZip AML, though not a basis for treatment change on its own.
Who should care
Limitations
Retrospective, with small mutation subgroups (the cytoplasmic-tail group in CEBPA-bZip was small) and many subgroup comparisons.
CSF3R mutation effect depends on molecular context and mutation class.
This is an AI-generated summary of the published abstract, not a clinical recommendation. It may not reflect the full text or subsequent updates โ verify against the original source before acting on it.
Abstract
Colony-stimulating factor 3 receptor (CSF3R) mutations occur in acute myeloid leukaemia (AML), but their prognostic significance remains unclear. We analysed 2500 patients with newly diagnosed de novo non-acute promyelocytic leukaemia (APL) AML treated with intensive induction therapy. CSF3Rmut were identified in 3.5% and frequently co-occurred with CCAAT/enhancer-binding protein alpha bZIP domain (CEBPA-bZip) mutations and RUNX family transcription factor 1::RUNX1 partner transcriptional co-repressor 1 (RUNX1::RUNX1T1) fusions. In the entire cohort, CSF3Rmut were associated with inferior relapse-free survival (RFS), but not overall survival (OS) or event-free survival (EFS). The prognostic impact was context-dependent. The adverse association was most consistent in CEBPA-bZip AML, with inferior OS, EFS (hazard ratio [HR], 2.76; pโ=โ0.001) and RFS (HR, 2.66; pโ=โ0.002) and persisted after multivariable adjustment and time-dependent modelling of transplantation. Membrane-proximal activating mutations (MPM) were associated primarily with inferior RFS (HR, 2.27; pโ=โ0.001) in the overall cohort, whereas cytoplasmic-tail truncating mutations (CTTM) showed no consistent adverse effect. In CEBPA-bZip AML, both MPM and CTTM showed adverse EFS and RFS associations, although the CTTM subgroup was small. The prognostic impact of CSF3Rmut in AML depends on molecular context and functional mutation class, with the strongest adverse associations observed in CEBPA-bZip AML.
DOI
10.1111/bjh.70909
PMID
42893900
Research metadata (titles, abstracts, authors, publication dates) is sourced from Europe PMC, which aggregates content from PubMed/MEDLINE, PubMed Central, and other providers under the U.S. National Library of Medicine (NLM/NCBI). NLM does not claim copyright on abstracts; the original journal publishers or authors may hold rights to the full articles. AI-generated summaries are derived only from the abstract, never the full text, and are always labeled as AI-generated.
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