Daunorubicin-Cytarabine Liposome (CPX-351) in the Management of Newly Diagnosed Secondary AML: A New Twist on an Old Cocktail

Publication date: Available online 13 May 2019Source: Best Practice & Research Clinical HaematologyAuthor(s): Joseph E. Maakaron, Alice S. MimsAbstractInitial therapy for acute myeloid leukemia (AML) remained stagnant for approximately four decades despite advances in improved understanding of pathogenesis and prognostication of the disease. The cornerstone of AML treatment has historically included three days of an anthracycline (daunorubicin or idarubicin) combined with continuous infusion of cytarabine for 7 days, the “7 + 3” regimen. Several attempts have been made to improve on this regimen by dose-intensification, altering routes of delivery, changing schedules of delivery and addition of other agents. However, despite modest improvements in response rates, overall survival has not typically been improved with the exception of the addition of midostaurin to 7+3 in FLT3 mutated AML (1-6). However, the re-vamping of the delivery of both daunorubicin and cytarabine in a liposomal encapsulation, known as CPX-351, did show improvements of overall survival compared to traditional 7+3 in newly diagnosed secondary and therapy-related AML in patients aged 60-75. This led to the Food and Drug Administration (FDA) approval of the agent for both of these subtypes of AML in August of 2017. Herein we will review the rationale and preclinical development of CPX-351 and discuss the pivotal studies that led to its FDA approval.
Source: Best Practice and Research Clinical Haematology - Category: Hematology Source Type: research