Secondary acute myeloid leukemia (sAML) poorly responds to conventional treatments and allogeneic stem cell transplantation (HSCT). We evaluated toxicity and efficacy of CPX-351 in 71 elderly patients (median age 66 years) with sAML enrolled in the Italian Named (Compassionate) Use Program. Sixty days treatment-related mortality was 7% (5/71). The response rate at the end of treatment was: CR/CRi in 50/71 patients (70.4%), PR in 6/71 (8.5%), and NR in 10/71 (19.7%). After a median follow-up of 11 months relapse was observed in 10/50 patients (20%) and 12 months cumulative incidence of relapse (CIR) was 23.6%. Median duration of response was not reached. In competing risk analysis, CIR was reduced when HSCT was performed in first CR (12 months CIR of 5% and 37.4%, respectively, for patients receiving (=20) or not (=30) HSCT, p = 0.012). Twelve-months OS was 68.6% (median not reached). In landmark analysis, HSCT in CR1 was the only significant predictor of longer survival (12 months OS of 100 and 70.5%, for patients undergoing or not HSCT in CR1, respectively, p = 0.011). In conclusion, we extend 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.

CPX-351 treatment in secondary acute myeloblastic leukemia is effective and improves the feasibility of allogeneic stem cell transplantation: results of the Italian compassionate use program / Guolo, F.; Fianchi, L.; Minetto, P.; Clavio, M.; Gottardi, M.; Galimberti, S.; Rizzuto, G.; Rondoni, M.; Bertani, G.; Dargenio, M.; Bilio, A.; Scappini, B.; Zappasodi, P.; Scattolin, A. M.; Grimaldi, F.; Pietrantuono, G.; Musto, P.; Cerrano, M.; D'Ardia, S.; Audisio, E.; Cignetti, A.; Pasciolla, C.; Pavesi, F.; Candoni, A.; Gurreri, C.; Morselli, M.; Alati, C.; Fracchiolla, N.; Rossi, G.; Caizzi, M.; Carnevale-Schianca, F.; Tafuri, A.; Rossi, G.; Ferrara, F.; Pagano, L.; Lemoli, R. M.. - In: BLOOD CANCER JOURNAL. - ISSN 2044-5385. - 10:10(2020), pp. 1-11. [10.1038/s41408-020-00361-8]

CPX-351 treatment in secondary acute myeloblastic leukemia is effective and improves the feasibility of allogeneic stem cell transplantation: results of the Italian compassionate use program

Candoni A.;
2020

Abstract

Secondary acute myeloid leukemia (sAML) poorly responds to conventional treatments and allogeneic stem cell transplantation (HSCT). We evaluated toxicity and efficacy of CPX-351 in 71 elderly patients (median age 66 years) with sAML enrolled in the Italian Named (Compassionate) Use Program. Sixty days treatment-related mortality was 7% (5/71). The response rate at the end of treatment was: CR/CRi in 50/71 patients (70.4%), PR in 6/71 (8.5%), and NR in 10/71 (19.7%). After a median follow-up of 11 months relapse was observed in 10/50 patients (20%) and 12 months cumulative incidence of relapse (CIR) was 23.6%. Median duration of response was not reached. In competing risk analysis, CIR was reduced when HSCT was performed in first CR (12 months CIR of 5% and 37.4%, respectively, for patients receiving (=20) or not (=30) HSCT, p = 0.012). Twelve-months OS was 68.6% (median not reached). In landmark analysis, HSCT in CR1 was the only significant predictor of longer survival (12 months OS of 100 and 70.5%, for patients undergoing or not HSCT in CR1, respectively, p = 0.011). In conclusion, we extend 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.
2020
10
10
1
11
CPX-351 treatment in secondary acute myeloblastic leukemia is effective and improves the feasibility of allogeneic stem cell transplantation: results of the Italian compassionate use program / Guolo, F.; Fianchi, L.; Minetto, P.; Clavio, M.; Gottardi, M.; Galimberti, S.; Rizzuto, G.; Rondoni, M.; Bertani, G.; Dargenio, M.; Bilio, A.; Scappini, B.; Zappasodi, P.; Scattolin, A. M.; Grimaldi, F.; Pietrantuono, G.; Musto, P.; Cerrano, M.; D'Ardia, S.; Audisio, E.; Cignetti, A.; Pasciolla, C.; Pavesi, F.; Candoni, A.; Gurreri, C.; Morselli, M.; Alati, C.; Fracchiolla, N.; Rossi, G.; Caizzi, M.; Carnevale-Schianca, F.; Tafuri, A.; Rossi, G.; Ferrara, F.; Pagano, L.; Lemoli, R. M.. - In: BLOOD CANCER JOURNAL. - ISSN 2044-5385. - 10:10(2020), pp. 1-11. [10.1038/s41408-020-00361-8]
Guolo, F.; Fianchi, L.; Minetto, P.; Clavio, M.; Gottardi, M.; Galimberti, S.; Rizzuto, G.; Rondoni, M.; Bertani, G.; Dargenio, M.; Bilio, A.; Scappini, B.; Zappasodi, P.; Scattolin, A. M.; Grimaldi, F.; Pietrantuono, G.; Musto, P.; Cerrano, M.; D'Ardia, S.; Audisio, E.; Cignetti, A.; Pasciolla, C.; Pavesi, F.; Candoni, A.; Gurreri, C.; Morselli, M.; Alati, C.; Fracchiolla, N.; Rossi, G.; Caizzi, M.; Carnevale-Schianca, F.; Tafuri, A.; Rossi, G.; Ferrara, F.; Pagano, L.; Lemoli, R. M.
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