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Venetoclax and azacitidine followed by allogeneic transplant results in excellent outcomes and may improve outcomes versus maintenance therapy among newly diagnosed AML patients older than 60

Abstract

The combination of venetoclax (ven) and azacitidine (aza) has resulted in high response rates in the upfront treatment of AML in patients age > 75 and patients unfit for intensive chemotherapy. Given the poor historical outcomes in patients age ≥ 60 treated with induction chemotherapy, ven/aza has become our institutional preference for the initial treatment of non-core binding factor (CBF) AML patients age ≥ 60. The benefit of allogeneic stem cell transplant (SCT) in patients who achieve response to ven/aza is uncertain. We report outcomes of SCT-eligible patients treated at our center. Between 1/2015 and 1/2020, 119 newly diagnosed non-CBF AML patients age ≥ 60 received ven/aza as initial therapy. 21 patients underwent SCT; 31 additional patients were potentially SCT eligible but deferred SCT. Overall survival (OS) was significantly greater among SCT patients (median survival not reached) versus potentially SCT eligible patients not undergoing SCT (median 518 days) (p = 0.01). Our data suggest that ven/aza followed by SCT in newly diagnosed AML patients older than ≥ 60 results in excellent outcomes and likely improves outcomes over maintenance therapy. Ongoing investigation will further refine the optimal timing of and selection of patients for SCT based on prognostic disease features and response assessments.

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Fig. 1: Patients and transplant guidelines.
Fig. 2: Overall survival.
Fig. 3: Selected additional overall survival outcomes.
Fig. 4: SCT outcomes.

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Acknowledgements

The authors thank all of the patients and the outstanding team at the Blood Disorders Center at the University of Colorado.

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D.P. and J.G. designed research, performed research, analyzed data, and wrote the paper. A.W., C.M., M.S., C.J., R.R. and C.S. analyzed data and wrote the paper.

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Correspondence to Jonathan A. Gutman.

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D.P. has received research funding and served as a consultant to Abbvie.

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Pollyea, D.A., Winters, A., McMahon, C. et al. Venetoclax and azacitidine followed by allogeneic transplant results in excellent outcomes and may improve outcomes versus maintenance therapy among newly diagnosed AML patients older than 60. Bone Marrow Transplant (2021). https://doi.org/10.1038/s41409-021-01476-7

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