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Multicenter Study
. 2025 Feb 25;9(4):752-758.
doi: 10.1182/bloodadvances.2024014279.

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

Thomas Cluzeau  1   2   3 , Fabio Guolo  4   5 , Edmond Chiche  1 , Paola Minetto  4 , Ramy Rahme  6 , Sarah Bertoli  7 , Luana Fianchi  8 , Jean-Baptiste Micol  9 , Michele Gottardi  10 , Pierre Peterlin  11 , Sara Galimberti  12 , Xavier Thomas  13 , Giuliana Rizzuto  14 , Olivier Legrand  15 , Michela Rondoni  16 , Emmanuel Raffoux  17 , Giambattista Bertani  18 , Alexis Caulier  19 , Michelina D'Argenio  20 , Caroline Bonmati  21 , Atto Billio  22 , Caroline Lejeune  23 , Barbara Scappini  24 , Arnaud Pigneux  25 , Patrizia Zappasodi  26 , Christan Recher  7 , Francesco Grimaldi  27 , Lionel Ades  6 , Roberto M Lemoli  4   5
Affiliations
Multicenter Study

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

Thomas Cluzeau et al. Blood Adv. .

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.

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Conflict of interest statement

Conflict-of-interest disclosure: T.C., R.M.L., and L.A. declare consultancy and speaker bureau fees from Jazz Pharmaceuticals. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Mutational profile of the cohort (number of mutations).
Figure 2.
Figure 2.
OS and MRD status.
Figure 3.
Figure 3.
OS in patients who underwent transplantation vs those who did not.
Figure 4.
Figure 4.
OS and MRD status in patients who underwent transplantation.
See this image and copyright information in PMC

References

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