CPX-351 results in similar overall survival (OS) as venetoclax plus azacitidine but is associated with more infections and hospitalizations in patients with acute myeloid leukemia (AML), according to a real-world effectiveness study 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 researchers wrote in their report.

The researchers analyzed data from 656 patients with newly diagnosed AML. At baseline, the median age was lower in the CPX-351 group, at 67 years, compared with 75 years in the venetoclax/azacitidine group (P <.001).

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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. The median OS was 13 months with CPX-351 and 11 months with venetoclax/azacitidine (hazard ratio [HR], 0.88; 95% CI, 0.71-1.08; P =.22). The 1-year OS rate was 51% with CPX-351 and 48% with venetoclax/azacitidine. The 2-year OS rate was 28% in both groups.

Responses were also similar between the groups. The rate of complete response with or without complete recovery of blood counts was 49% with CPX-351 and 43% with venetoclax/azacitidine.

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

However, CPX-351 was associated with a higher rate of infection than venetoclax/azacitidine — 67% and 36%, respectively (P =.0004). Febrile neutropenia was seen in 90% of the CPX-351 group and 54% of the venetoclax/azacitidine group (P <.00005).

The mean length of inpatient hospital stay was longer with CPX-351 than with venetoclax/azacitidine — 41 days and 15 days, respectively (P <.00005).

“Although subject to ascertainment and misclassification bias risks, key adverse events, including length of stay, febrile neutropenia, and infections, appear to favor treatment with ven/aza,” the researchers wrote. “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 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

This article originally appeared on Hematology Advisor