CPX-351 first-line therapy shows 61% overall response in therapy-related AML or AML with myelodysplastic-related changes.

This retrospective monocentric cohort study included 60 consecutive patients treated with CPX-351 as first-line therapy for therapy-related AML or AML with myelodysplastic-related changes. The setting was not reported. The primary outcome assessed determinants of response, relapse, and survival, while secondary outcomes included overall response rate, complete response, relapse rate, relapse-free survival, and overall survival.

The overall response rate was 61%, and the complete response rate was 58%. Among patients who achieved a complete response, the relapse rate was 50%. The median relapse-free survival was 154 days, and the median overall survival was 412 days. In univariable analysis, leukocytosis at diagnosis, complex karyotype, and mutations of IDH1 and SRSF2 were correlated with lower survival. In multivariable analysis, leukocytosis and complex karyotype retained statistical significance. Additionally, the presence of a complex karyotype at diagnosis predicted lower response rates.

Safety and tolerability were described as having an excellent safety profile, though specific adverse events, serious adverse events, discontinuations, and follow-up duration were not reported. Key limitations include the retrospective monocentric design, small sample size of 60 patients, and lack of reported safety data. These factors limit the certainty of the findings and their direct applicability to broader clinical practice.