Long-Term Real-World Efficacy of CPX-351 Therapy for Patients With High-Risk AML
Among patients with high-risk therapy-related acute myeloid leukemia (t-AML) and AML with myelodysplasia-related changes (MRC-AML), treatment with CPX-351 induction therapy yielded achievement of measurable residual disease (MRD) status which was significantly associated with improved overall survival (OS), according to retrospective study results published in Blood Advances.
CPX-351, a liposomal formulation of daunorubicin and cytarabine, has been approved for the treatment of t-AML and MRC-AML, but long-term real-world data on MRD outcomes associated with CPX-351 use have been limited. Researchers conducted this retrospective study to evaluate the clinical efficacy of CPX-351 in a real-world setting with long-term outcomes, and evaluate the impact of MRD.
Overall, 168 patients with t-AML (28%) or MRC-AML (72%) who received 1 (n = 91) or 2 (n = 11) cycles of CPX-351 were included. The median age was 66 years (range, 20 to 83). In terms of genetic risk, 84% of patients had adverse risk according to ELN 2017 classification, with 36% of patients at intermediate risk and 5% at favorable risk. About 21% of patients had TP53 mutation, 18% RUNX1, and 13% had ASXL1.
The overall response rate (ORR) was 60% among all patients, with a complete response (CR) of 53%. MRD below 10⁻³ was achieved by 65% of patients. At 3 years, the median OS was 13.34 months (range, 9.35 to 17.33). Patients who had MRD levels below 10⁻³ (20.4 months) had longer OS compared to patients above10⁻³ (12.9 months, P = .03). In multivariate analysis, MRD > 10⁻³ was the only independent predictor of lower OS (hazard ratio [HR], 2.6; 95% confidence interval [CI], 1.2 to 5.5; P = .013).
Among patients who underwent allogeneic hematopoietic stem cell transplantation (HSCT; n = 60) while MRD-negative, median OS was higher than patients were MRD-positive (not reached vs. 26.0 months; P = .06).
“After 3 years of follow-up, the improved OS with CPX-351 was confirmed in the real-life setting,” the researchers concluded, adding, “achievement of MRD negativity contributed to the improvement of OS in the overall population and, maybe, in patients who received transplant.”
Source:
Cluzeau T, Fabio Guolo, Chiche E, et al. Long-term real world evidence of CPX-351 of high-risk AML patients identified high rate of negative MRD and prolonged OS. Blood Advances. Published online February 12, 2025. doi: 10.1182/bloodadvances.2024014279
