1. Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia.
- Author
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Rautenberg C, Stölzel F, Röllig C, Stelljes M, Gaidzik V, Lauseker M, Kriege O, Verbeek M, Unglaub JM, Thol F, Krause SW, Hänel M, Neuerburg C, Vucinic V, Jehn CF, Severmann J, Wass M, Fransecky L, Chemnitz J, Holtick U, Schäfer-Eckart K, Schröder J, Kraus S, Krüger W, Kaiser U, Scholl S, Koch K, Henning L, Kobbe G, Haas R, Alakel N, Röhnert MA, Sockel K, Hanoun M, Platzbecker U, Holderried TAW, Morgner A, Heuser M, Sauer T, Götze KS, Wagner-Drouet E, Döhner K, Döhner H, Schliemann C, Schetelig J, Bornhäuser M, Germing U, Schroeder T, and Middeke JM
- Subjects
- Adult, Aged, Aged, 80 and over, Allografts, Disease-Free Survival, Female, Follow-Up Studies, Humans, Male, Middle Aged, Neoplasm, Residual, Survival Rate, Cytarabine administration & dosage, Daunorubicin administration & dosage, Hematopoietic Stem Cell Transplantation, Leukemia, Myeloid, Acute blood, Leukemia, Myeloid, Acute mortality, Leukemia, Myeloid, Acute therapy
- Abstract
To investigate the efficacy and toxicities of CPX-351 outside a clinical trial, we analyzed 188 patients (median age 65 years, range 26-80) treated for therapy-related acute myeloid leukemia (t-AML, 29%) or AML with myelodysplasia-related changes (AML-MRC, 70%). Eighty-six percent received one, 14% two induction cycles, and 10% received consolidation (representing 22% of patients with CR/CRi) with CPX-351. Following induction, CR/CRi rate was 47% including 64% of patients with available information achieving measurable residual disease (MRD) negativity (<10
-3 ) as measured by flow cytometry. After a median follow-up of 9.3 months, median overall survival (OS) was 21 months and 1-year OS rate 64%. In multivariate analysis, complex karyotype predicted lower response (p = 0.0001), while pretreatment with hypomethylating agents (p = 0.02) and adverse European LeukemiaNet 2017 genetic risk (p < 0.0001) were associated with lower OS. Allogeneic hematopoietic cell transplantation (allo-HCT) was performed in 116 patients (62%) resulting in promising outcome (median survival not reached, 1-year OS 73%), especially in MRD-negative patients (p = 0.048). With 69% of patients developing grade III/IV non-hematologic toxicity following induction and a day 30-mortality of 8% the safety profile was consistent with previous findings. These real-world data confirm CPX-351 as efficient treatment for these high-risk AML patients facilitating allo-HCT in many patients with promising outcome after transplantation., (© 2021. The Author(s).)- Published
- 2021
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