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Real World Study on the Best CPX-351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation
Fabio Guolo1,2, Luana Fianchi3, Maria Paola Martelli4
1University of Genoa, Department of Internal Medicine (DiMI), Genoa, Italy.
Insights
CPX-351 improves outcomes in secondary acute myeloid leukemia (s-AML). Allogeneic stem cell transplant (allo-HSCT) is beneficial, especially when performed early after achieving remission. Patients not undergoing transplant benefit from completing all CPX-351 cycles.
Area of Science:
- Hematology
- Oncology
- Clinical Trials
Background:
- Secondary acute myeloid leukemia (s-AML) has poorer outcomes compared to de novo AML.
- CPX-351 demonstrated superiority over conventional 3+7 chemotherapy in a registration trial for s-AML.
- Optimal treatment duration, timing of allogeneic stem cell transplantation (allo-HSCT), and CPX-351 activity in s-AML subgroups require further investigation.
Purpose of the Study:
- To retrospectively analyze the outcomes of s-AML patients treated with CPX-351.
- To evaluate the impact of treatment duration, timing of allo-HSCT, and specific patient subgroups on CPX-351 efficacy.
- To identify factors influencing overall survival (OS) in s-AML patients receiving CPX-351.
Main Methods:
- Retrospective analysis of 513 s-AML patients treated with CPX-351.
- Assessment of complete remission (CR) rates after induction and consolidation cycles.
- Evaluation of allo-HSCT utilization, consolidation cycle completion, and their association with OS.
Main Results:
- The CR rate was 58% after induction, increasing to 66% after cycle 2.
- 48.8% of patients received allo-HSCT, associated with significantly longer OS (not reached vs. 16.3 months).
- Patients with NPM1 mutations or ELN 2017 favorable risk had longer OS. Completing CPX-351 cycles benefited only non-transplanted patients.
Conclusions:
- CPX-351 is effective in s-AML, with CR rates improving after a second cycle.
- Early allo-HSCT upon achieving CR is recommended for eligible s-AML patients.
- Complete CPX-351 treatment courses benefit patients not proceeding to transplant, while early transplant is key for others.
Abstract:
In the registration clinical trial 301 (NCT01696084), CPX-351 has shown to be superior to conventional 3 + 7 in secondary AML (s-AML). However, the optimal duration of treatment, the best timing for allogeneic stem cell transplantation (allo-HSCT), and the activity of CPX-351 in specific s-AML subgroups are unclear. To evaluate these aspects, a total of 513 s-AML patients (median age 65.6 years, 19-79) treated with CPX-351 were retrospectively analyzed. Complete remission (CR) rate after induction was 297/513 (58%), increasing to 340/513 (66%) after cycle 2. Among the 340 responding patients, 118 (34.7%), 137 (40.3%), and 85 (25%) received none, one, or two consolidation cycles of CPX-351, respectively. Overall, 230/513 patients (48.8%) received allo-HSCT. Median follow up was 23.66 months and median overall survival (OS) was 16.23 months. Patients with mutated NPM1 or with ELN 2017 favorable risk (p < 0.05) had a significantly longer OS (p < 0.05). In a landmark analysis, receiving allo-HSCT was associated with a longer survival (Median OS not reached vs. 16.3 months for patients receiving or not receiving allo-HSCT, p < 0.05). Completion of all allowed CPX-351 cycles was beneficial only in patients not proceeding to transplant (p < 0.05), whereas in transplanted patients additional CPX-351 cycles did not improve outcome. Our analysis suggests that also s-AML patients with NPM1 mutations and those belonging to the ELN 2017 favorable risk category benefit from CPX-351. In eligible patients, allo-HSCT should be performed as soon as a CR is achieved, whereas patients not undergoing transplant benefit from a complete CPX-351 schedule.
