CPX-351 (also known as VYXEOS® or Vyxeos liposomal) is a dual-drug liposomal encapsulation of cytarabine and daunorubicin in a synergistic 5:1 molar ratio and was the first example that utilized CombiPlex®, a combination drug technology platform. Superior efficacy with CPX-351 in the pivotal phase 3 randomized clinical trial versus its conventional free-drug counterpart led to its approval for the treatment of newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes in multiple countries. Emerging evidence indicates that CPX-351 affords additional benefits compared with conventional chemotherapy, including protection against intestinal dysbiosis and fungal colonization, fewer infectious complications, and a lower incidence of cardiotoxicity. This review examines the mechanisms underlying CPX-351’s therapeutic effects and highlights its expanding role in AML treatment by summarizing efficacy and safety data from preclinical models, the pivotal clinical trial, and real-world studies. Particular focus is given to recent findings on CPX-351’s intestinal and cardioprotective properties, which together strengthen its safety and efficacy profile compared with conventional chemotherapy.

The Role of CPX-351 in the Acute Myeloid Leukemia Treatment Landscape: Mechanism of Action, Efficacy, and Safety / L. Pagano, R. Danesi, E. Benedetti, R. Morgagni, L. Romani, A. Venditti. - In: DRUGS. - ISSN 0012-6667. - (2025), pp. e2300229.1-e2300229.12. [Epub ahead of print] [10.1007/s40265-025-02194-w]

The Role of CPX-351 in the Acute Myeloid Leukemia Treatment Landscape: Mechanism of Action, Efficacy, and Safety

R. Danesi
;
2025

Abstract

CPX-351 (also known as VYXEOS® or Vyxeos liposomal) is a dual-drug liposomal encapsulation of cytarabine and daunorubicin in a synergistic 5:1 molar ratio and was the first example that utilized CombiPlex®, a combination drug technology platform. Superior efficacy with CPX-351 in the pivotal phase 3 randomized clinical trial versus its conventional free-drug counterpart led to its approval for the treatment of newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes in multiple countries. Emerging evidence indicates that CPX-351 affords additional benefits compared with conventional chemotherapy, including protection against intestinal dysbiosis and fungal colonization, fewer infectious complications, and a lower incidence of cardiotoxicity. This review examines the mechanisms underlying CPX-351’s therapeutic effects and highlights its expanding role in AML treatment by summarizing efficacy and safety data from preclinical models, the pivotal clinical trial, and real-world studies. Particular focus is given to recent findings on CPX-351’s intestinal and cardioprotective properties, which together strengthen its safety and efficacy profile compared with conventional chemotherapy.
Settore BIOS-11/A - Farmacologia
2025
10-mag-2025
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/1171873
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