CPX-351, a novel liposomal formulation of cytarabine and daunorubicin, represents the standard of care in fit patients with acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) and therapy-related AML (t-AML). Considering its better safety profile than conventional intensive chemotherapy, we investigated its cost-to-benefit ratio, in terms of overall survival and of mortality, in a large multicentric series of AML-MRC and t-AML receiving CPX-351 outside clinical trials between 2019 and 2022. Patients were classified as fit or unfit for intensive chemotherapy through a comprehensive evaluation of age, comorbidities, and performance status by adopting Italian Society of Hematology/Italian Society of Experimental Hematology/Gruppo Italiano per il Trapianto di Midollo Osseo (SIE/SIES/GITMO) criteria. Disease risk was defined according to the European LeukemiaNet 2017 classification. Before treatment start, 328 of 403 (81.4%) patients were classified as fit and 75 of 403 (18.6%) as unfit. Three hundred and ninety-six had a full genetic/cytogenetic profile, with 17 (4%) being categorized as favorable risk, 162 (41%) intermediate risk, and 217 (55%) adverse risk according to European LeukemiaNet 2017. After induction, 230 of 403 (57.1%) patients achieved complete remission, with no differences between fit (57.3%) and unfit (56%) patients. However, the 2 groups significantly differed in terms of survival (median overall survival, 18 months vs 8 months for fit and unfit patients, respectively) and of 28- and 100-day mortality (4.6% vs 10.7% at 28 days and 14.3% vs 32% at 100 days for fit and unfit patients, respectively). In conclusion, the SIE/SIES/GITMO criteria distinguished patient subgroups with different short- and long-term outcomes after treatment with CPX-351. The update or design of dedicated fitness criteria could represent a future and valid strategy to optimize the use of this specific treatment.
Impact of fitness categorization according to SIE/SIES/GITMO criteria in therapy-related and AML-MRC receiving CPX-351 / Palmieri, Raffaele; Guolo, Fabio; Fianchi, Luana; Ferrara, Felicetto; Minetto, Paola; Martelli, Maria Paola; Riva, Carola; Chiusolo, Patrizia; Rondoni, Michela; Capria, Saveria; Minotti, Clara; Pilo, Federica; Perrone, Salvatore; Corbingi, Andrea; Grimaldi, Francesco; De Luca, Giulia; Fili, Carla; Alati, Caterina; Mannelli, Francesco; Lessi, Federica; Marchesi, Francesco; Brunetti, Lorenzo; Capelli, Debora; Lina Piccioni, Anna; Vetro, Calogero; Erika Palumbo, Fanny; Mazzone, Carla; Sperotto, Alessandra; Luzi, Giovanni; Dargenio, Michela; Mariani, Sabrina; Cerrano, Marco; Mulè, Antonino; Di Veroli, Ambra; Meddi, Elisa; Moretti, Federico; Papayannidis, Cristina; Isidori, Alessandro; Ferrari, Antonella; Galimberti, Sara; Cilloni, Daniela; Breccia, Massimo; Lanza, Francesco; Gottardi, Michele; Cairoli, Roberto; Candoni, Anna; Pagano, Livio; Todisco, Elisabetta; Massimo Lemoli, Roberto; Venditti, Adriano; Buccisano, Francesco. - In: BLOOD ADVANCES. - ISSN 2473-9537. - (2026).
Impact of fitness categorization according to SIE/SIES/GITMO criteria in therapy-related and AML-MRC receiving CPX-351
Raffaele Palmieri;Maria Paola Martelli;Saveria Capria;Salvatore Perrone;Francesco Grimaldi;Giulia De Luca;Sabrina Mariani;Elisa Meddi;Federico Moretti;Antonella Ferrari;Massimo Breccia;
2026
Abstract
CPX-351, a novel liposomal formulation of cytarabine and daunorubicin, represents the standard of care in fit patients with acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) and therapy-related AML (t-AML). Considering its better safety profile than conventional intensive chemotherapy, we investigated its cost-to-benefit ratio, in terms of overall survival and of mortality, in a large multicentric series of AML-MRC and t-AML receiving CPX-351 outside clinical trials between 2019 and 2022. Patients were classified as fit or unfit for intensive chemotherapy through a comprehensive evaluation of age, comorbidities, and performance status by adopting Italian Society of Hematology/Italian Society of Experimental Hematology/Gruppo Italiano per il Trapianto di Midollo Osseo (SIE/SIES/GITMO) criteria. Disease risk was defined according to the European LeukemiaNet 2017 classification. Before treatment start, 328 of 403 (81.4%) patients were classified as fit and 75 of 403 (18.6%) as unfit. Three hundred and ninety-six had a full genetic/cytogenetic profile, with 17 (4%) being categorized as favorable risk, 162 (41%) intermediate risk, and 217 (55%) adverse risk according to European LeukemiaNet 2017. After induction, 230 of 403 (57.1%) patients achieved complete remission, with no differences between fit (57.3%) and unfit (56%) patients. However, the 2 groups significantly differed in terms of survival (median overall survival, 18 months vs 8 months for fit and unfit patients, respectively) and of 28- and 100-day mortality (4.6% vs 10.7% at 28 days and 14.3% vs 32% at 100 days for fit and unfit patients, respectively). In conclusion, the SIE/SIES/GITMO criteria distinguished patient subgroups with different short- and long-term outcomes after treatment with CPX-351. The update or design of dedicated fitness criteria could represent a future and valid strategy to optimize the use of this specific treatment.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
