Determinants of survival in patients treated with CPX-351 for acute myeloid leukemia

CPX-351 for the treatment of acute myeloid leukemia (AML) has demonstrated better efficacy compared with standard chemotherapy regimens, with an excellent safety profile. We aimed to assess the determinants of response, relapse, and survival in patients treated with CPX-351 as the first-line therapy for AML. In this retrospective monocentric study, we analyzed 60 consecutive patients treated with CPX-351 for therapy-related AML or AML with myelodysplastic-related changes. We observed a 61% overall response rate, with 58% complete response. The presence of complex karyotype at diagnosis predicted lower response rates. Among patients who achieved complete response, the relapse rate was 50%, with a median relapse-free survival of 154 days. The median overall survival was 412 days. We found that the biological profile at diagnosis is associated with survival. Leukocytosis at diagnosis, complex karyotype, and mutations of IDH1 and SRSF2 were correlated with lower survival in the univariable analysis. In the multivariable analysis, leukocytosis and complex karyotype retained their statistical significance.