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.
Real World Study on the Best CPX-351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation / Guolo, F.; Fianchi, L.; Martelli, M. P.; Lussana, F.; Grimaldi, F.; Pilo, F.; Rondoni, M.; Fili, C.; Minetto, P.; Capelli, D.; Chiusolo, P.; Breccia, M.; Mastaglio, S.; Bernardi, M.; Bocchia, M.; Fumagalli, M.; Galimberti, S.; Mancini, V.; Piccioni, A. L.; Maurillo, L.; Fracchiolla, N. S.; Palmieri, R.; Vetro, C.; Papayannidis, C.; Brunetti, L.; Sperotto, A.; Gigli, F.; Zappasodi, P.; Mule, A.; Patti, C.; Borlenghi, E.; Dargenio, M.; Lessi, F.; Cerrano, M.; Cilloni, D.; Isidori, A.; Lunghi, M.; Alati, C.; Gurrieri, C.; Riva, C.; Marconi, G.; Lotesoriere, I.; Gatani, S.; Scattolin, A. M.; Caizzi, M.; Perrone, S.; Billio, A.; Gherlinzoni, F.; Mannelli, F.; Gottardi, M.; Cairoli, R.; Candoni, A.; Ferrara, F.; Pagano, L.; Lemoli, R. M.; Venditti, A.; Todisco, E.. - In: AMERICAN JOURNAL OF HEMATOLOGY. - ISSN 0361-8609. - 100:12(2025), pp. 2293-2304. [10.1002/ajh.70083]
Real World Study on the Best CPX-351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation
Capelli D.;Brunetti L.;
2025-01-01
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
