Real-life experience with CPX-351 and impact on the outcome of high-risk AML patients: a multicentric French cohort

CPX-351 is a liposomal formulation of cytarabine and daunorubicin approved for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (MRC-AML). We retrospectively analyzed the efficacy and safety of CPX-351 in a real-world setting in 103 patients from 12 French centers, including the evaluation of molecular abnormalities at baseline and minimal residual disease (MRD) in responding patients, compared with a historical data set from Bordeaux-Toulouse DATAML registry. A favorable safety profile was observed, with a low frequency of alopecia (11%) and gastrointestinal toxicity (50%). The overall response rate after induction was 59%, and MRD,1023 was achieved in 57% of complete response (CR)/CR with incomplete hematological recovery (CRi) patients. Only the presence of mutated TP53 (P = .02) or PTPN11 (P = .004) predicted lower response in multivariate analysis. Interestingly, high-risk molecular prognosis subgroups defined by 2017 European LeukemiaNet risk stratification, including ASXL1 and RUNX1 mutations, were not associated with a significantly lower response rate using CPX-351. With amedian follow-up of 8.6 months, median overall survival (OS) was 16.1 months. Thirty-six patients underwent allogeneic stem cell transplantation with a significantly longer median OS compared with nontransplanted patients (P<.001). In multivariate analyses, only spliceosome mutations were associated with better OS (P=.04). In comparison with intensive chemotherapy, there was no difference in OS for patients,60 years. These data confirm the efficacy and safety of CPX-351 in high-risk AML(t-AML and MRC-AML) in a real-life setting. CPX-351 is a treatment of choice for patients aged >60 years.

Automated summary

This study retrospectively analyzed the efficacy and safety of CPX-351 in 103 patients from 12 French centers, finding a favorable safety profile and a high overall response rate. Presence of high-risk molecular prognosis subgroups did not significantly impact response to CPX-351, while spliceosome mutations were associated with better overall survival.