Published 2021
| Version v1
Journal article
Real-life experience with CPX-351 and impact on the outcome of high-risk AML patients: a multicentric French cohort
Creators
- Chiche, Edmond
- Rahme, Ramy
- Bertoli, Sarah
- Dumas, Pierre-Yves
- Micol, Jean-Baptiste
- Hicheri, Yosr
- Pasquier, Florence
- Peterlin, Pierre
- Chevallier, Patrice
- Thomas, Xavier
- Loschi, Michael
- Genthon, Alexis
- Legrand, Ollivier
- Mohty, Mohamad
- Raffoux, Emmanuel
- Auberger, Patrick
- Caulier, Alexis
- Joris, Magalie
- Bonmati, Caroline
- Roth-Guepin, Gabrielle
- Lejeune, Caroline
- Pigneux, Arnaud
- Vey, Norbert
- Recher, Christian
- Ades, Lionel
- Cluzeau, Thomas
Contributors
Others:
- Université Côte d'Azur - Faculté de Médecine (UCA Faculté Médecine) ; Université Côte d'Azur (UCA)
- Centre Hospitalier Universitaire de Nice (CHU Nice)
- Hopital Saint-Louis [AP-HP] (AP-HP) ; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)
- Institut Universitaire du Cancer de Toulouse - Oncopole (IUCT Oncopole - UMR 1037) ; Université Toulouse III - Paul Sabatier (UT3) ; Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-CHU Toulouse [Toulouse]-Institut National de la Santé et de la Recherche Médicale (INSERM)
- CHU Toulouse [Toulouse]
- CHU Bordeaux [Bordeaux]
- Institut Gustave Roussy (IGR)
- Université Paris-Saclay
- Département d'hématologie [Gustave Roussy] ; Institut Gustave Roussy (IGR)
- Institut Paoli-Calmettes ; Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)
- Centre hospitalier universitaire de Nantes (CHU Nantes)
- Centre Hospitalier Lyon Sud [CHU - HCL] (CHLS) ; Hospices Civils de Lyon (HCL)
- Centre méditerranéen de médecine moléculaire (C3M) ; Université Nice Sophia Antipolis (1965 - 2019) (UNS) ; COMUE Université Côte d'Azur (2015-2019) (COMUE UCA)-COMUE Université Côte d'Azur (2015-2019) (COMUE UCA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Côte d'Azur (UCA)
- CHU Saint-Antoine [AP-HP] ; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)
- Centre de Recherche Saint-Antoine (CRSA) ; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)
- CHU Amiens-Picardie
- Centre Hospitalier Régional Universitaire de Nancy (CHRU Nancy)
- Institut de Cancérologie Lucien Neuwirth ; CHU Saint-Etienne
- Université Paris Cité (UPCité)
Description
CPX-351 is a liposomal formulation of cytarabine and daunorubicin approved for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (MRC-AML). We retrospectively analyzed the efficacy and safety of CPX-351 in a real-world setting in 103 patients from 12 French centers, including the evaluation of molecular abnormalities at baseline and minimal residual disease (MRD) in responding patients, compared with a historical data set from Bordeaux-Toulouse DATAML registry. A favorable safety profile was observed, with a low frequency of alopecia (11%) and gastrointestinal toxicity (50%). The overall response rate after induction was 59%, and MRD,1023 was achieved in 57% of complete response (CR)/CR with incomplete hematological recovery (CRi) patients. Only the presence of mutated TP53 (P = .02) or PTPN11 (P = .004) predicted lower response in multivariate analysis. Interestingly, high-risk molecular prognosis subgroups defined by 2017 European LeukemiaNet risk stratification, including ASXL1 and RUNX1 mutations, were not associated with a significantly lower response rate using CPX-351. With amedian follow-up of 8.6 months, median overall survival (OS) was 16.1 months. Thirty-six patients underwent allogeneic stem cell transplantation with a significantly longer median OS compared with nontransplanted patients (P<.001). In multivariate analyses, only spliceosome mutations were associated with better OS (P=.04). In comparison with intensive chemotherapy, there was no difference in OS for patients,60 years. These data confirm the efficacy and safety of CPX-351 in high-risk AML(t-AML and MRC-AML) in a real-life setting. CPX-351 is a treatment of choice for patients aged >60 years.
Abstract
International audienceAdditional details
Identifiers
- URL
- https://hal-u-picardie.archives-ouvertes.fr/hal-03605963
- URN
- urn:oai:HAL:hal-03605963v1