1. 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
- Author
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Paola Minetto, Carmela Gurreri, Fabio Guolo, Anna Candoni, Giovanni Rossi, Giambattista Bertani, Marco Cerrano, Patrizia Zappasodi, Francesco Grimaldi, Atto Bilio, Anna Maria Scattolin, Barbara Scappini, Pellegrino Musto, Marino Clavio, Agostino Tafuri, Livio Pagano, Crescenza Pasciolla, Giuseppe Pietrantuono, Monica Morselli, Alessandro Cignetti, Roberto M. Lemoli, Sara Galimberti, Ernesta Audisio, Nicola Stefano Fracchiolla, Fabrizio Carnevale-Schianca, Felicetto Ferrara, Stefano D'Ardia, Giuseppe Rossi, Francesca Pavesi, Manuela Caizzi, Michele Gottardi, Luana Fianchi, Giuliana Rizzuto, Michela Rondoni, Michela Dargenio, Caterina Alati, 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., Ferrara, F., Pagano, L., and Lemoli, R. M.
- Subjects
Compassionate Use Trials ,Male ,medicine.medical_specialty ,medicine.medical_treatment ,neoplasms ,acute myeloid - leukemia ,minimal residual disease ,myelodysplastic syndrome ,molar ratiotherapy, neoplasms, cytarabine, daunorubicin ,Drug development ,Hematopoietic stem cell transplantation ,Gene mutation ,lcsh:RC254-282 ,Disease-Free Survival ,Article ,molar ratiotherapy ,Internal medicine ,Medicine ,Secondary Acute Myeloid Leukemia ,Humans ,Cumulative incidence ,Survival rate ,Aged ,Leukemia ,business.industry ,Mortality rate ,Daunorubicin ,Cytarabine ,Hematopoietic Stem Cell Transplantation ,Neoplasms, Second Primary ,Hematology ,Middle Aged ,lcsh:Neoplasms. Tumors. Oncology. Including cancer and carcinogens ,Allografts ,Transplantation ,Survival Rate ,Settore MED/15 - MALATTIE DEL SANGUE ,Regimen ,Leukemia, Myeloid, Acute ,Combination drug therapy ,Oncology ,Italy ,Female ,business ,Follow-Up Studies - 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.
- Published
- 2020