CPX-351 in acute myeloid leukemia: can a new formulation maximize the efficacy of old compounds?
Abstract
INTRODUCTION: The management of Acute Myeloid Leukemia (AML) (with the exception of acute promyelocytic leukemia) has remained largely unchanged over the past 40 years. In particular, patients defined as high-risk, according to the 2017 European Leukemia Net recommendations, represent a subgroup with poor response to current therapies that are frequently associated with high-grade toxicity and potentially fatal complications. Areas covered: Preliminary results from an ongoing phase III clinical trial suggest that CPX-351 could represent an interesting treatment option in both induction and 'bridge-to-transplant' settings. In particular, 60- to 75-year-old patients with secondary AML, when treated with CPX-351, exhibit superior overall survival (HR = 0.69; P = 0.005; median OS 9.56 vs. 5.95 months), event free survival (HR = 0.74; P = 0.021), and composite response rate (47.7% vs. 33.3%; P = 0.016) as compared to standard '7 + 3' therapy. Herein, we detail the main pharmacological features of CPX-351 and review updated results of clinical trials investigating its employment in AML. Expert commentary: Novel liposome-based drugs display a high therapeutic index and represent a promising alternative to unencapsulated drugs, especially when high-risk features complicate the use of standard treatments. Further efforts in both understanding AML biology and improving nanodrug design are needed.
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CPX-351 is approved by the US FDA for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) with myelodysplasia-related changes (AML-MRC) and therapy-related AML (t-AML).
“This liposomal nanoparticles are approved from US FDA for the for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) with myelodysplasia-related changes (AML-MRC) and therapy-related AML (t-AML).347”
From Smart nanoparticles for cancer therapy (2023) · cites this paper as [347]
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- Authors
- Brunetti, C., Anelli, L., Zagaria, A., Specchia, G., & Albano, F.
- Affiliations
- University of Bari Aldo Moro (IT)
- Corresponding author
- Albano, Francesco
- Venue
- Expert Review of Hematology
- Publisher
- Informa UK Limited
- Published
- 2017-08-24
- Volume
- 10
- Issue
- 10
- Pages
- 853-862
- ISSN
- 1747-4086 (ISSN-L)
- 1747-4094
- OpenAlex
- W2747821992
- Cited by
- 23
- References
- 73
- Citations by year
- 2024: 3 · 2023: 2 · 2022: 1 · 2021: 1 · 2020: 3 · 2019: 8 · 2018: 5
- FWCI
- 0.69
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Expert Review of Hematology (publishedVersion)
- PubMed (publishedVersion)
- CINECA IRIS Institutional Research Information System (University of Bari Aldo Moro) (submittedVersion)
- Topics
- Health Sciences › Medicine › Hematology › Acute Myeloid Leukemia Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinoids in leukemia and cellular processes
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Keywords
- CPX-351, acute myeloid leukemia, high-risk AML, liposomal doxorubicin, secondary acute myeloid leukemia, 7+3 induction chemotherapy, overall survival, event-free survival
- MeSH terms
- Animals, Antineoplastic Agents, Antineoplastic Combined Chemotherapy Protocols, Drug Compounding, Drug Evaluation, Preclinical, Humans, Liposomes, Leukemia, Myeloid, Acute, Treatment Outcome, Clinical Trials, Phase I as Topic, Clinical Trials, Phase II as Topic, Clinical Trials, Phase III as Topic, Nanomedicine, Drug Discovery
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