TY - JOUR
T1 - Real-life experience with CPX-351 and impact on the outcome of high-risk AML patients
T2 - A multicentric French cohort
AU - Chiche, Edmond
AU - Rahme, Ramy
AU - Bertoli, Sarah
AU - Dumas, Pierre Yves
AU - Micol, Jean Baptiste
AU - Hicheri, Yosr
AU - Pasquier, Florence
AU - Peterlin, Pierre
AU - Chevallier, Patrice
AU - Thomas, Xavier
AU - Loschi, Michael
AU - Genthon, Alexis
AU - Legrand, Ollivier
AU - Mohty, Mohamad
AU - Raffoux, Emmanuel
AU - Auberger, Patrick
AU - Caulier, Alexis
AU - Joris, Magalie
AU - Bonmati, Caroline
AU - Roth-Guepin, Gabrielle
AU - Lejeune, Caroline
AU - Pigneux, Arnaud
AU - Vey, Norbert
AU - Recher, Christian
AU - Ades, Lionel
AU - Cluzeau, Thomas
N1 - Publisher Copyright:
© 2021 by The American Society of Hematology.
PY - 2021/1/12
Y1 - 2021/1/12
N2 - 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 profilewas 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 5.02) or PTPN11 (P 5.004) 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 (P5.04). In comparisonwith 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.
AB - 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 profilewas 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 5.02) or PTPN11 (P 5.004) 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 (P5.04). In comparisonwith 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.
UR - http://www.scopus.com/inward/record.url?scp=85099175137&partnerID=8YFLogxK
U2 - 10.1182/bloodadvances.2020003159
DO - 10.1182/bloodadvances.2020003159
M3 - Article
C2 - 33570629
AN - SCOPUS:85099175137
SN - 2473-9529
VL - 5
SP - 176
EP - 184
JO - Blood Advances
JF - Blood Advances
IS - 1
ER -