Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS

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.

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Publication Details

Journal
Blood Advances
Published
2024-10-25
DOI
https://doi.org/10.1182/bloodadvances.2024014279
Citations
14
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
6.36
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article

Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS

Sara Galimberti, Giuliana Rizzuto, Arnaud Pigneux, Francesco Grimaldi et al.
14 citations
Blood Advances
Acute Myeloid Leukemia Research
6.36
article

Long-term real-world evidence of CPX-351 of high-risk patients with AML identified high rate of negative MRD and prolonged OS

Sara Galimberti, Giuliana Rizzuto, Arnaud Pigneux, Francesco Grimaldi, Fabio Guolo, Lionel Adès, Jean‐Baptiste Micol, Roberto Massimo Lemoli, Christian Récher, Michelina Dargenio, Michela Rondoni, Michele Gottardi, Caroline Bonmati, Pierre Péterlin, Barbara Scappini, Giambattista Bertani, Sarah Bertoli, Luana Fianchi, Patrizia Zappasodi, Emmanuel Raffoux, Ramy Rahmé, Edmond Chiche, Paola Minetto, Xavier Thomas, Thomas Cluzeau, Alexis Caulier, Atto Billio, Olivier Legrand, Caroline Lejeune
article en
14 citations

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.

Blood AdvancesVol. 9(4)
University of Pisa (IT), Boston Children's Hospital (US), Inserm (FR), Université Paris Cité (FR), Institut Gustave Roussy (FR), Agostino Gemelli University Polyclinic (IT), Centre Hospitalier Universitaire de Bordeaux (FR), Centre Hospitalier Universitaire de Nice (FR), Centre Hospitalier Universitaire de Nantes (FR), Centre Hospitalier Universitaire de Toulouse (FR), Sorbonne Université (FR), Ospedale di Bolzano (IT), Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (IT), Centre Hospitalier Universitaire Amiens-Picardie (FR), Institut universitaire du cancer de Toulouse Oncopole (FR), Centre Hospitalier Régional et Universitaire de Nancy (FR), Hôpital Saint-Antoine (FR), Ospedale Papa Giovanni XXIII (IT), Azienda Unità Sanitaria Locale Della Romagna (IT), Istituto Oncologico Veneto (IT), Azienda Ospedaliero-Universitaria Careggi (IT), Federico II University Hospital (IT), Centre Méditerranéen de Médecine Moléculaire (FR), Hôpital Saint-Louis (FR), Ospedale Policlinico San Martino (IT), Boston Children's Museum (US), Policlinico San Matteo Fondazione (IT), Ospedale Vito Fazzi (IT), Centre Hospitalier Universitaire de Saint-Étienne (FR), Azienda Ospedaliera Universitaria Pisana (IT), University of Genoa (IT)
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Openalex Percentile: Top 3%
Acute Myeloid Leukemia Research
6.36
DOIOpen Access PDFOpenAlex
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