CPX-351 can provide long-term survival benefits over conventional chemotherapy in older patients with newly diagnosed acute myeloid leukemia (AML), according to results published in The Lancet Haematology.

In a randomized, phase 3 trial (ClinicalTrials.gov Identifier: NCT01696084), researchers compared CPX-351 to 7+3 chemotherapy (cytarabine for 7 days and daunorubicin for 3 days) in adults aged 60 to 75 years with newly diagnosed, high-risk or secondary AML.

The study enrolled 309 patients, and they were randomly assigned to receive CPX-351 (n=153) or 7+3 chemotherapy (n=156). Patients received up to 2 induction cycles and could proceed to hematopoietic stem cell transplant (HSCT) at the investigator’s discretion. In all, 35% of patients in the CPX-351 arm and 25% of those in the 7+3 arm underwent HSCT.

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Among HSCT recipients, 25% in the CPX-351 arm and 46% in the 7+3 arm received additional therapy after study treatment and before HSCT. The median time from randomization to HSCT was 114 days in both arms.

At the final analysis, the median follow-up was 60.91 months for the CPX-351 arm and 59.93 months for the 7+3 arm.

Overall survival (OS) was superior in the CPX-351 arm, both in the overall cohort and among HSCT recipients.

For the entire cohort, the median OS was 9.33 months in the CPX-351 arm and 5.95 months in the 7+3 arm (hazard ratio [HR], 0.70; 95% CI, 0.55-0.91). The 5-year OS was 18% and 8%, respectively.

Among HSCT recipients, the median OS was not reached in the CPX-351 arm and was 10.25 months in the 7+3 arm (HR, 0.51; 95% CI, 6.21-16.69). The 3-year OS was 56% and 23%, respectively.

Progressive leukemia was the most common cause of death, accounting for 56% of deaths in the CPX-351 arm and 53% of deaths in the 7+3 arm. In both arms, 14% of deaths were due to adverse events.

“Together, these results support the previous evidence that CPX-351 has the ability to contribute to long-term remission and survival in patients aged 60-75 years with newly diagnosed, high-risk or secondary acute myeloid leukemia and further support the use of CPX-351 in this patient population,” the study authors wrote.

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


Lancet JE, Uy GL, Newell LF, et al. CPX-351 versus 7+3 cytarabine and daunorubicin chemotherapy in older adults with newly diagnosed high-risk or secondary acute myeloid leukaemia: 5-year results of a randomised, open-label, multicentre, phase 3 trial. Lancet Haematol. 2021;8(7):e481-e491. doi:10.1016/S2352-3026(21)00134-4