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ASCO 2025 | Real-world effectiveness and safety of CPX-351 in patients with secondary AML

Eunice Wang, MD, Roswell Park Comprehensive Cancer Center, Buffalo, NY, comments on the real-world effectiveness and safety of CPX-351 in patients with secondary acute myeloid leukemia (AML). Dr Wang notes that despite initial concerns, CPX-351 has been found to retain its efficacy in the real-world setting, with tolerability also being demonstrated. Notably, the drug has been successfully administered in outpatient settings, a novel approach for induction chemotherapy in AML. This interview took place during the 2025 American Society of Clinical Oncology (ASCO) Meeting in Chicago, IL.

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Transcript

CPX-351 is a liposomal cytarabine daunorubicin formulation, which was approved a few years ago for patients with therapy-related and MDS-related AML. We however have been struggling to say, is it really effective for patients, particularly younger patients under the age of 60, who were enrolled in that randomized Phase III trial, and what is the real-world usefulness of this drug? 

So very encouragingly, we partnered with a few medical centers to look at our own experience in the post-hoc, post-approval setting, and we were surprised to see that this drug retains its efficacy even in the real-world setting...

CPX-351 is a liposomal cytarabine daunorubicin formulation, which was approved a few years ago for patients with therapy-related and MDS-related AML. We however have been struggling to say, is it really effective for patients, particularly younger patients under the age of 60, who were enrolled in that randomized Phase III trial, and what is the real-world usefulness of this drug? 

So very encouragingly, we partnered with a few medical centers to look at our own experience in the post-hoc, post-approval setting, and we were surprised to see that this drug retains its efficacy even in the real-world setting. The drug was tolerable. And most importantly, we were able to show that in some centers, this drug has even been given in the outpatient setting, which is novel for induction chemotherapy for acute myeloid leukemia. So overall, the real-world efficacy report really substantiated the initial findings of its efficacy in this patient population, as well as its tolerability and safety, even out into academic and or community centers.

 

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