Summary CPX‐351 is a standard front‐line induction regimen for newly diagnosed acute myeloid leukaemia (AML) with myelodysplasia‐related changes (AML‐MRC). The 2022 International Consensus Classification (ICC) and World Health Organization (WHO) classifications redefine AML with myelodysplasia‐related (AML‐MR) to include myelodysplasia‐related mutations as well as cytogenetic abnormalities. Clinical outcomes of patients treated with CPX‐351 within these refined AML‐MR classifications remain unclear. We conducted a retrospective, multicentre study of 235 adults with newly diagnosed AML‐MR treated with CPX‐351 across seven US academic centres. Patients were stratified by age (younger: <60 vs. older: ≥60 years) and AML‐MR subgroup: cytogenetics (AML‐MRc), molecular (AML‐MRm) and antecedent haematological disorder (AML‐AHD). Outcomes included complete remission (CR) and CR with incomplete recovery (CR/CRi), rates of allogeneic haematopoietic stem cell transplant (alloHSCT) and overall survival (OS). The overall CR/CRi rate of CPX‐351 was 52%, with no difference by age. AML‐MRm had the highest CR/CRi rate (57%). Among CR/CRi responders, 55% underwent alloHSCT (<60 years: 53% vs. ≥60 years: 57%). Median OS was 13.8 months with no significant difference by age. Younger AML‐MRm patients had longer median OS compared with older AML‐MRm patients (38.0 vs. 19.5 months; p = 0.05). Favourable outcomes in AML‐MRm, particularly in younger patients, support molecular classification in guiding therapy and selectively extending CPX‐351 use beyond older adults.
Peters et al. (2025) studied this question.