In a real-world effectiveness study, CPX-351 resulted in similar overall survival (OS) as venetoclax plus azacitidine but was associated with more infections and hospitalization among patients with acute myeloid leukemia (AML), according to the results published in Blood Advances.

“In the absence of prospective randomized comparisons of these regimens, we used retrospective observational data to evaluate various outcomes of patients with newly diagnosed AML receiving either CPX-351 or venetoclax/azacitidine,” the authors wrote in their report.

The retrospective study evaluated data from 656 patients with newly diagnosed AML from the electronic health record databases of Flatiron Health or the University of Pennsylvania. The primary outcome was OS and secondary outcomes included remission, remission duration, and event-free survival.

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At baseline, the median age was lower in the CPX-351 group at 67 compared with 75 in the venetoclax/azacitidine group (P <.001). More patients in the venetoclax/azacitidine group were treated in the community setting (P <.001), had de novo disease (P <.001), and used Medicare or other insurance (P =.047). Other characteristics, such as high-risk mutations, FLT3, IDH1/2, or NPM1 status, and comorbidity index were similar.

There was no significant difference in OS between the treatment groups, with a median of 13 months with CPX-351 compared with 11 months for venetoclax/azacitidine (hazard ratio [HR], 0.88; 95% CI, 0.71-1.08; P =.22). The 1- and 2-year OS was 51% and 28% with CPX-351, respectively, compared with 48% and 28% with venetoclax/azacitidine.

The response rate was also similar, with 49% and 43% of patients achieving a complete response with or without complete recovery of blood counts in the CPX-351 or venetoclax/azacitidine groups, respectively.

Relapse-free survival was a median of 11 months with CPX-351 compared with 16 months with venetoclax/azacitidine.

Mortality during treatment, both at 30 days and 60 days, was similar between the treatment groups. The 30-day mortality was 5% with either CPX-351 or venetoclax/azacitidine, and the 60-day mortality was 10% and 13%, respectively.

Treatment with CPX-351 was associated with higher rates of infection at 67% compared with 36% of patients in the venetoclax/azacitidine group (P =.0004). Febrile neutropenia was seen in 90% of the CPX-351 group compared with 54% of the venetoclax/azacitidine group (P <.00005).

Any hospital admission and length of stay before the next treatment cycle was longer with CPX-351 at a median of 41 days compared with 15 days venetoclax/azacitidine (P <.00005).

The authors concluded, “in older adults with AML, initial treatment with CPX-351 or venetoclax/azacitidine resulted in similar OS.” They added, “This raises the question of whether intensive chemotherapy should remain the standard recommendation for fit older patients or even younger patients with adverse genetics.”

Disclosures: Some of the study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.


Matthews AH, Perl AE, Luger SM, et al. Real-world effectiveness of CPX-351 vs venetoclax and azacitidine in acute myeloid leukemia. Blood Adv. 2022;6:3997-4005. doi: 10.1182/bloodadvances.2022007265