Secondary acute myeloid leukemia poorly responds to conventional treatments and allogeneic stem cell transplantation (HSCT). The purpose of the study was to evaluate the toxicity and efficacy of CPX-351 in 71 elderly patients with sAML enrolled in the Italian Named Use Program.

60 total days of treatment-related mortality was 7%. The response rate at the end of treatment was: CR/CRi in 70.4%patients, PR in 8.5%, and NR in 19.7%. After a median follow-up of 11 months, relapse was observed in 10/50 patients and in 1 year the cumulative incidence of relapse was 23.6%. The median duration of response was not reached. In competing for risk analysis, CIR was reduced when HSCT was performed in the first CR. Twelve-months OS was 68.6%. In landmark analysis, HSCT in CR1 was the only significant predictor of longer survival.

The study concluded through its findings that it extends to a real-life setting, the notion that CPX is an effective regimen for high-risk AML patients and may improve the results of HSCT.

Reference: https://www.nature.com/articles/s41408-020-00361-8