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Master Thesis

Expérience en vie réelle du CPX-351 dans les LAM à haut risque : résultats d’une cohorte multicentrique française

Abstract : 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. 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 <10-3 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 additional sex comb-like 1 (ASXL1) and runt-related transcription factor 1 (RUNX1) mutations, were not associated with a significantly lower response rate using CPX-351. With a median 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). 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.
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Master Thesis
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Contributor : Bibliothèque Universitaire de Médecine Nice Connect in order to contact the contributor
Submitted on : Friday, May 7, 2021 - 12:05:34 PM
Last modification on : Thursday, November 25, 2021 - 3:16:40 AM


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  • HAL Id : dumas-03220570, version 1


Edmond Chiche. Expérience en vie réelle du CPX-351 dans les LAM à haut risque : résultats d’une cohorte multicentrique française. Médecine humaine et pathologie. 2021. ⟨dumas-03220570⟩



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