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

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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Main Authors: 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
Format: Article
Language:English
Published: Elsevier 2025-02-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952924006293
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Summary: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.
ISSN:2473-9529

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