Real World Study on the Best <scp>CPX</scp> ‐351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation

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.

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Publication Details

Journal
American Journal of Hematology
Published
2025-09-24
DOI
https://doi.org/10.1002/ajh.70083
Citations
3
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
5.31

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article

Real World Study on the Best CPX ‐351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation

Sara Galimberti, Monica Bocchia, Antonino Mulè, Anna Candoni et al.
3 citations
American Journal of Hematology
Acute Myeloid Leukemia Research
5.31
article

Real World Study on the Best CPX ‐351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation

Sara Galimberti, Monica Bocchia, Antonino Mulè, Anna Candoni, Valentina Mancini, Francesco Grimaldi, Fabio Guolo, Filíppo Gherlinzoni, Anna Maria Scattolin, Roberto Massimo Lemoli, Michelina Dargenio, Federica Pilo, Nicola Fracchiolla, Michela Rondoni, Elisabetta Todisco, Michele Gottardi, Alessandra Sperotto, Roberto Cairoli, Daniela Cilloni, Giovanni Marconi, Erika Borlenghi, Federica Lessi, Luca Maurillo, Massimo Bernardi, Francesco Mannelli, Calogero Vetro, Manuela Caizzi, Sara Mastaglio, Patrizia Chiusolo, Marco Cerrano, Felicetto Ferrara, Carmela Gurrieri, Cristina Papayannidis, Livio Pagano, Luana Fianchi, Ivana Lotesoriere, Federico Lussana, Monia Lunghi, Patrizia Zappasodi, Debora Capelli, Raffaele Palmieri, Alessandro Isidori, Paola Minetto, Caterina Patti, Lorenzo Brunetti, Anna Lina Piccioni, Federica Gigli, Carola Riva, Salvatore Perrone, Maria Paola Martelli, Massimo Breccia, Atto Billio, Samuele Gatani, Monica Fumagalli, Caterina Alati, Carla Filì, Adriano Venditti
article en
3 citations

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.

American Journal of HematologyVol. 100(12)
Università Cattolica del Sacro Cuore (IT), University of Pisa (IT), University of Rome Tor Vergata (IT), University of Modena and Reggio Emilia (IT), Università degli Studi del Piemonte Orientale “Amedeo Avogadro” (IT), University of Padua (IT), University of Perugia (IT), Azienda Ospedaliera San Giovanni Addolorata (IT), Agostino Gemelli University Polyclinic (IT), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT), Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT), Ospedale Antonio Cardarelli (IT), Azienda Ospedaliera di Perugia (IT), Ospedale di Bolzano (IT), Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (IT), Azienda ospedaliera "Bianchi-Melacrino-Morelli" (IT), Ospedale Vincenzo Cervello (IT), Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT), Ospedale Papa Giovanni XXIII (IT), Ospedali Riuniti di Ancona (IT), Azienda Unità Sanitaria Locale Della Romagna (IT), Istituto Oncologico Veneto (IT), Azienda Ospedaliera San Gerardo (IT), Azienda Ospedaliero-Universitaria Careggi (IT), Ospedale Santa Maria (IT), Istituto di Ematologia di Bologna (IT), Azienda Ospedaliero Universitaria Maggiore della Carita (IT), Ospedale Santa Maria della Misericordia di Udine (IT), Ospedale di Circolo di Busto Arsizio (IT), Centro di Riferimento Oncologico (IT), A. O. Ordine Mauriziano di Torino (IT), Policlinico Universitario di Catania (IT), Azienda Ospedaliera Universitaria Senese (IT), Stem Cell Institute (PA), Ospedale Santa Maria Goretti (IT), Ospedale dell' Angelo (IT), Azienda Ospedaliera Ospedale Maggiore (IT), Ca' Foncello Hospital (IT), Ospedale Policlinico San Martino (IT), Policlinico San Matteo Fondazione (IT), Ospedale Vito Fazzi (IT), Istituti di Ricovero e Cura a Carattere Scientifico (IT), Azienda Sanitaria Universitaria Integrata di Trieste (IT), European Institute of Oncology (IT), University of Naples Federico II (IT), University of Genoa (IT), Sapienza University of Rome (IT)
Università degli Studi di Padova, Università degli Studi di Genova, Università di Pisa, Istituto Oncologico Veneto, Dipartimento di Medicina e Chirurgia, Università degli Studi di Milano-Bicocca
Openalex Percentile: Top 4%
Acute Myeloid Leukemia Research
5.31
DOIOpen Access PDFOpenAlex
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