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Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia.

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.

SUBMITTER: Rautenberg C

PROVIDER: S-EPMC8490353 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature

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Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia.

Rautenberg Christina C Stölzel Friedrich F Röllig Christoph C Stelljes Matthias M Gaidzik Verena V Lauseker Michael M Kriege Oliver O Verbeek Mareike M Unglaub Julia Marie JM Thol Felicitas F Krause Stefan W SW Hänel Mathias M Neuerburg Charlotte C Vucinic Vladan V Jehn Christian-Friedrich CF Severmann Julia J Wass Maxi M Fransecky Lars L Chemnitz Jens J Holtick Udo U Schäfer-Eckart Kerstin K Schröder Josephine J Kraus Sabrina S Krüger William W Kaiser Ulrich U Scholl Sebastian S Koch Kathrin K Henning Lea L Kobbe Guido G Haas Rainer R Alakel Nael N Röhnert Maximilian-Alexander MA Sockel Katja K Hanoun Maher M Platzbecker Uwe U Holderried Tobias A W TAW Morgner Anke A Heuser Michael M Sauer Tim T Götze Katharina S KS Wagner-Drouet Eva E Döhner Konstanze K Döhner Hartmut H Schliemann Christoph C Schetelig Johannes J Bornhäuser Martin M Germing Ulrich U Schroeder Thomas T Middeke Jan Moritz JM

Blood cancer journal 20211004 10

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 informat ...[more]

PMID: 34608129

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Project description:BackgroundLAMVYX was a multicenter, single-arm, phase 2 trial designed to validate the safety and efficacy of CPX-351 in patients aged 60-75 years with newly diagnosed, secondary acute myeloid leukemia and to generate evidence on key issues not addressed in the preceding regulatory pivotal trial.MethodsThe primary end point of the study was the complete remission (CR)/CR with incomplete hematologic recovery (CRi) rate after induction. Eligible patients were recommended to undergo allogeneic hematopoietic stem cell transplantation after the first consolidation cycle. Alternatively, patients could undergo up to six maintenance cycles with CPX-351.ResultsTwenty-nine patients (49%; 95% exact confidence interval [CI], 37%-62%) patients achieved a CR/CRi after one or two cycles of induction, with a measurable residual disease negativity rate of 67% as assessed by centralized, multiparameter flow cytometry. Among patients who had serial next-generation sequencing analyses available, clearance of somatic mutations that were present at diagnosis was achieved in 7 (35%). The median follow-up among survivors was 16.8 months (range, 8.7-24.3 months). The median event-free survival was 3.0 months (95% CI, 1.4-7.3 months), and the median overall survival was 7.4 months (95% CI, 3.7-12.7 months). In landmark analyses at day +100 from diagnosis, the 1-year overall and event-free survival rate among patients who underwent allogeneic hematopoietic stem cell transplantation was 70% (95% CI, 47%-100%) and 70% (95% CI, 47%-100%), respectively. The corresponding values were 89% (95% CI, 71%-100%) and 44% (95% CI, 21%-92%), respectively, for patients who entered the maintenance phase. No significant longitudinal changes were observed in severity index or quality-of-life visual analog scale scores.ConclusionsThe current data provide novel insights that might inform the clinical positioning and optimal use of CPX-351, complementing previous results (ClinicalTrials.gov identifier NCT04230239).

Dataset Information

Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia.

Publications

Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia.

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