Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS
Abstract: CPX-351 has been approved for patients with therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (MRC-AML). No extensive data are available on measurable residual disease (MRD) and long-term clinical outcome using CPX-351 in AML in real life. We retrosp...
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Elsevier
2025-02-01
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| Series: | Blood Advances |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2473952924006293 |
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| author | Thomas Cluzeau Fabio Guolo Edmond Chiche Paola Minetto Ramy Rahme Sarah Bertoli Luana Fianchi Jean-Baptiste Micol Michele Gottardi Pierre Peterlin Sara Galimberti Xavier Thomas Giuliana Rizzuto Olivier Legrand Michela Rondoni Emmanuel Raffoux Giambattista Bertani Alexis Caulier Michelina D’Argenio Caroline Bonmati Atto Billio Caroline Lejeune Barbara Scappini Arnaud Pigneux Patrizia Zappasodi Christan Recher Francesco Grimaldi Lionel Ades Roberto M. Lemoli |
| author_facet | Thomas Cluzeau Fabio Guolo Edmond Chiche Paola Minetto Ramy Rahme Sarah Bertoli Luana Fianchi Jean-Baptiste Micol Michele Gottardi Pierre Peterlin Sara Galimberti Xavier Thomas Giuliana Rizzuto Olivier Legrand Michela Rondoni Emmanuel Raffoux Giambattista Bertani Alexis Caulier Michelina D’Argenio Caroline Bonmati Atto Billio Caroline Lejeune Barbara Scappini Arnaud Pigneux Patrizia Zappasodi Christan Recher Francesco Grimaldi Lionel Ades Roberto M. Lemoli |
| author_sort | Thomas Cluzeau |
| collection | DOAJ |
| description | Abstract: CPX-351 has been approved for patients with therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (MRC-AML). No extensive data are available on measurable residual disease (MRD) and long-term clinical outcome using CPX-351 in AML in real life. We retrospectively collected data from 168 patients in 36 centers in France and Italy who had received 1 or 2 cycles of induction with CPX-351. All patients were aged >18 years and had newly diagnosed, untreated t-AML or MRC-AML. With a median follow-up of 3 years, the median overall survival (OS) was 13.3 months. The median OS was 20.4 months vs 12.9 months for patients with MRD below or above 10–3, respectively (P = .006). In a multivariate analysis, only MRD >10–3 was associated with a poorer OS (hazard ratio, 2.6; 95% confidence interval, 1.2-5.5; P = .013). We also observed a trend toward a better median OS in patients who underwent hematopoietic stem cell transplantation with MRD <10–3 (not reached vs 26.0 months; P = .06). Achievement of MRD negativity contributed to the improvement of OS in the overall population and, maybe, in patients receiving transplant. These data provide the rationale for the 2 ongoing studies evaluating CPX-351 vs 7+3 in non–MRC-AML and non–t-AML using MRD as the primary end point for ALFA-2101 phase 2 clinical trial and event-free survival for AMLSG 30-18 phase 3 clinical trial. |
| format | Article |
| id | doaj-art-b5e33f120f7745d5a751ce23af99dbb7 |
| institution | Kabale University |
| issn | 2473-9529 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Blood Advances |
| spelling | doaj-art-b5e33f120f7745d5a751ce23af99dbb72025-02-12T05:31:36ZengElsevierBlood Advances2473-95292025-02-0194752758Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OSThomas Cluzeau0Fabio Guolo1Edmond Chiche2Paola Minetto3Ramy Rahme4Sarah Bertoli5Luana Fianchi6Jean-Baptiste Micol7Michele Gottardi8Pierre Peterlin9Sara Galimberti10Xavier Thomas11Giuliana Rizzuto12Olivier Legrand13Michela Rondoni14Emmanuel Raffoux15Giambattista Bertani16Alexis Caulier17Michelina D’Argenio18Caroline Bonmati19Atto Billio20Caroline Lejeune21Barbara Scappini22Arnaud Pigneux23Patrizia Zappasodi24Christan Recher25Francesco Grimaldi26Lionel Ades27Roberto M. Lemoli28Hematology Department, Cote d’Azur University, Centre Hospitalier Universitaire Nice, Nice, France; Hematology Department, Nice University Hospital, Nice, France; Hematology Department Cote d’Azur University, Mediterranean Center for Molecular Medicine, Nice, France; Correspondence: Thomas Cluzeau, Hematology Department, Nice University Hospital, 151 route Saint Antoine de Ginestière, 06200 Nice, France;Department of Internal Medicine, Clinic of Hematology, University of Genoa, Genoa, Italy; Department of Hematology and Oncology, Istituto di Ricovero e Cura a Carattere Scientifico Ospedale Policlinico San Martino, Genoa, ItalyHematology Department, Cote d’Azur University, Centre Hospitalier Universitaire Nice, Nice, FranceDepartment of Internal Medicine, Clinic of Hematology, University of Genoa, Genoa, ItalySenior Hematology Department, Hospital Saint-Louis, Université Paris Cité, INSERM U944, Paris, FranceHematology Department, Institut universitaire du cancer de Toulouse-Oncopole, Centre Hospitalier Universitaire Toulouse, Toulouse, FranceHematology Department Istituto di Ematologia, Fondazione Policlinico Universitario A. Gemelli, Istituto di Ricovero e Cura a Carattere Scientifico, Rome, ItalyHematology Department, Institut Gustave Roussy, Paris, FranceHematology Department, Istituto di Ricovero e Cura a Carattere Scientifico Istituto Oncologico Veneto, Castelfranco Veneto, ItalyHematology Department, Centre Hospitalier Universitaire Nantes, Nantes, FranceOspedaliera Universitaria Ematologia, Dipartimento di Medicina Clinica e Sperimentale, Università di Pisa, Pisa, ItalyHematology Department, Hopitaux civils de Lyon, Centre Hospitalier Universitaire Lyon, Lyon, FranceHematology and Bone Marrow Transplant Unit, Azienda Ospedaliera Universitaria Papa Giovanni XXIII, Bergamo, ItalyHematology Department, Hopital Saint-Antoine, Paris, FranceUnità Operativa di Cancerologia di Ematologia, Azienda Unità Sanitaria Locale della Romagna, Ravenna, ItalyAdult Hematology Department, Hopital Saint-Louis, Paris, FranceHematology Department, S.C. Ematologia, ASST Grande Ospedale Metropolitano, Niguarda Ca' Granda Milano, Milan, ItalyDepartment of Hematology, Centre Hospitalier Universitaire Amiens, Amiens, FranceHematology Department, Ospedale Vito Fazzi, Lecce, ItalyHematology Department, centre hospitalier régional universitaire Nancy, Nancy, FranceHematology Department, Ospedale di Bolzano, Bolzano, ItalyHematology Department, L'institut de Cancérologie de la Loire, centre hospitalier universitaire Saint-Etienne, Saint-Etienne, FranceDipartimento di Oncologia-SODc Ematologia, Azienda Ospedaliero - Universitaria Careggi, Florence, ItalyHematology Department, centre hospitalier universitaire Bordeaux, Bordeaux, FranceHematology Department, Clinica Ematologica, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, ItalyHematology Department, Institut universitaire du cancer de Toulouse-Oncopole, Centre Hospitalier Universitaire Toulouse, Toulouse, FranceDipartimento di Medicina Clinica e Chirurgia, Azienda Ospedaliera Universitaria Federico II di Napoli, Naples, ItalySenior Hematology Department, Hospital Saint-Louis, Université Paris Cité, INSERM U944, Paris, FranceDepartment of Internal Medicine, Clinic of Hematology, University of Genoa, Genoa, Italy; Department of Hematology and Oncology, Istituto di Ricovero e Cura a Carattere Scientifico Ospedale Policlinico San Martino, Genoa, ItalyAbstract: CPX-351 has been approved for patients with therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (MRC-AML). No extensive data are available on measurable residual disease (MRD) and long-term clinical outcome using CPX-351 in AML in real life. We retrospectively collected data from 168 patients in 36 centers in France and Italy who had received 1 or 2 cycles of induction with CPX-351. All patients were aged >18 years and had newly diagnosed, untreated t-AML or MRC-AML. With a median follow-up of 3 years, the median overall survival (OS) was 13.3 months. The median OS was 20.4 months vs 12.9 months for patients with MRD below or above 10–3, respectively (P = .006). In a multivariate analysis, only MRD >10–3 was associated with a poorer OS (hazard ratio, 2.6; 95% confidence interval, 1.2-5.5; P = .013). We also observed a trend toward a better median OS in patients who underwent hematopoietic stem cell transplantation with MRD <10–3 (not reached vs 26.0 months; P = .06). Achievement of MRD negativity contributed to the improvement of OS in the overall population and, maybe, in patients receiving transplant. These data provide the rationale for the 2 ongoing studies evaluating CPX-351 vs 7+3 in non–MRC-AML and non–t-AML using MRD as the primary end point for ALFA-2101 phase 2 clinical trial and event-free survival for AMLSG 30-18 phase 3 clinical trial.http://www.sciencedirect.com/science/article/pii/S2473952924006293 |
| spellingShingle | Thomas Cluzeau Fabio Guolo Edmond Chiche Paola Minetto Ramy Rahme Sarah Bertoli Luana Fianchi Jean-Baptiste Micol Michele Gottardi Pierre Peterlin Sara Galimberti Xavier Thomas Giuliana Rizzuto Olivier Legrand Michela Rondoni Emmanuel Raffoux Giambattista Bertani Alexis Caulier Michelina D’Argenio Caroline Bonmati Atto Billio Caroline Lejeune Barbara Scappini Arnaud Pigneux Patrizia Zappasodi Christan Recher Francesco Grimaldi Lionel Ades Roberto M. Lemoli Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS Blood Advances |
| title | Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS |
| title_full | Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS |
| title_fullStr | Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS |
| title_full_unstemmed | Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS |
| title_short | Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS |
| title_sort | long term real world evidence of cpx 351 of high risk patients with aml identified high rate of negative mrd and prolonged os |
| url | http://www.sciencedirect.com/science/article/pii/S2473952924006293 |
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