The Clinical Safety and Efficacy of Cytarabine and Daunorubicin Liposome (CPX‐351) in Acute Myeloid Leukemia Patients: A Systematic Review

ABSTRACT Background Acute myeloid leukemia (AML) is an aggressive blood cancer with a poor prognosis when treated using conventional chemotherapy. CPX‐351, a liposomal formulation of cytarabine and daunorubicin, has demonstrated potential as a novel therapeutic strategy. Aim This systematic review aims to evaluate the clinical safety and efficacy of CPX‐351 compared to standard induction chemotherapy in patients with AML. Methods A systematic literature search was conducted using Web of Science, PubMed, Google Scholar, Ovid MEDLINE, and the Cochrane Library from 2011 to 2023. Overall, 14 clinical trials with more than 800 AML patients were included. Data on adverse events, survival outcomes, and response rates were extracted and analyzed. Results CPX‐351 exhibited an adverse event profile that was comparable to that of conventional chemotherapy. It resulted in complete remission rates of 18%–41% in relapsed/refractory AML patients. For newly diagnosed individuals, CPX‐351 led to complete remission rates of 41%–58%, surpassing the 14%–40% associated with standard chemotherapy. Additionally, it extended overall survival by 3.6–5.7 months, with significant advantages noted in high‐risk cytogenetics and secondary AML cases. Conclusion This review presents strong evidence supporting CPX‐351 as a therapeutic alternative with superior efficacy and comparable safety to standard chemotherapy across diverse AML populations. This represents a breakthrough in therapy, with demonstrated efficacy in AML cases.