{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T16:31:34Z","timestamp":1790699494703,"version":"4.1.0"},"reference-count":30,"publisher":"American Society of Hematology","issue":"1","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2021,1,12]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>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 &amp;lt;10\u22123 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 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 &amp;lt; .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 &amp;lt;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 \u226560 years.<\/jats:p>","DOI":"10.1182\/bloodadvances.2020003159","type":"journal-article","created":{"date-parts":[[2021,1,10]],"date-time":"2021-01-10T21:10:11Z","timestamp":1610313011000},"page":"176-184","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":107,"title":["Real-life experience with CPX-351 and impact on the outcome of high-risk AML patients: a multicentric French cohort"],"prefix":"10.1182","volume":"5","author":[{"given":"Edmond","family":"Chiche","sequence":"first","affiliation":[{"name":"Cote d\u2019Azur University, Hematology Department, Centre Hospitalier Universitaire de Nice, Nice, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ramy","family":"Rahm\u00e9","sequence":"additional","affiliation":[{"name":"Senior Hematology Department, Hospital Saint-Louis, Assistance Publique\u2013Hopitaux de Paris, Paris, 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