Real-world experience with CPX-351 in high-risk acute myeloid leukemia

CPX-351, a dual-drug liposomal encapsulation of daunorubicin/cytarabine, was approved for newly diagnosed therapy-related acute myeloid leukemia (AML) and AML with myelodysplasia-related changes in adults in 2017 (US; updated to patients aged ≥1 year in 2021) and 2018 (EU/UK) based on improved survival and remission and comparable safety versus 7 + 3 chemotherapy in a randomized trial in older adults. Real-world studies have since evaluated CPX-351 in routine practice across several countries and addressed important data gaps (e.g., use in younger adults, measurable residual disease negativity, outcomes by mutation). This review discusses real-world studies of CPX-351 as AML treatment, with the aim of helping prescribers make informed treatment decisions. • Findings from real-world studies of CPX-351 were generally consistent with RCT data. • Majority of real-world studies reported longer OS and higher or similar CR/CRi. • Comparable/higher proportions of patients bridged to HCT in real-world studies. • In real-world studies, 38–64% of patients achieved MRD negativity with CPX-351. • CPX-351 had low early mortality rates in the real-world with no new safety signals.