This pilot study evaluated CPX-351 in adults with newly diagnosed, favourable-intermediate risk, FLT3-ITD-negative AML. Twenty patients received CPX-351 for induction, with six also receiving gemtuzumab ozogamicin (GO). The complete response rate was 95%, with 42% achieving flow-based minimal residual disease (MRD) negativity post-induction. The 18-month leukaemia-free and overall survival estimates were 80% and 95% respectively. Adding GO appeared safe without prolonged cytopenias. Subclinical cardiotoxicity was observed in 25% of patients. The study demonstrated CPX-351's feasibility, with response and MRD-negativity rates comparable to standard '7 + 3' induction.
Keywords: AML; CPX‐351; MRD; cardiotoxicity; gemtuzumab ozogamicin; vyxeos.
© 2024 The Author(s). British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.