TY - JOUR
T1 - 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
T2 - Blood Cancer Journal
AU - Guolo, F.
AU - Fianchi, L.
AU - Minetto, P.
AU - Clavio, M.
AU - Gottardi, M.
AU - Galimberti, S.
AU - Rizzuto, G.
AU - Rondoni, M.
AU - Bertani, G.
AU - Dargenio, M.
AU - Bilio, A.
AU - Scappini, B.
AU - Zappasodi, P.
AU - Scattolin, A.M.
AU - Grimaldi, F.
AU - Pietrantuono, G.
AU - Musto, P.
AU - Cerrano, M.
AU - D’Ardia, S.
AU - Audisio, E.
AU - Cignetti, A.
AU - Pasciolla, C.
AU - Pavesi, F.
AU - Candoni, A.
AU - Gurreri, C.
AU - Morselli, M.
AU - Alati, C.
AU - Fracchiolla, N.
AU - Rossi, G.
AU - Caizzi, M.
AU - Carnevale-Schianca, F.
AU - Tafuri, A.
AU - Ferrara, F.
AU - Pagano, L.
AU - Lemoli, R.M.
N1 - Export Date: 12 October 2020
Correspondence Address: Guolo, F.; IRCCS Ospedale Policlinico San MartinoItaly; email: fabio.guolo21@gmail.com
PY - 2020
Y1 - 2020
N2 - 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.
AB - 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.
U2 - 10.1038/s41408-020-00361-8
DO - 10.1038/s41408-020-00361-8
M3 - Article
VL - 10
JO - Blood Cancer J.
JF - Blood Cancer J.
SN - 2044-5385
IS - 10
ER -