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Venetoclax is safe and tolerable as post-transplant maintenance therapy for AML patients at high risk for relapse

Bone Marrow Transplantation volume 58, pages 849–854 (2023)Cite this article

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Abstract

Relapse is the most common cause of mortality in acute myeloid leukemia (AML) patients after allogeneic stem cell transplant (SCT). Post-SCT maintenance strategies that prevent relapse are desirable but must be well tolerated and convenient to administer. We hypothesized single agent venetoclax (ven) may be an effective maintenance therapy among high relapse risk patients. Between February 2019 and December 2021, we administered post-SCT ven maintenance to 49 AML patients at high-risk for relapse as a prospectively defined off-label practice at our institution. Ven was planned to be administered until 1-year post-SCT. While temporary interruptions were common (67.3% of all patients), of those with >1 year follow up, 22/25 (88%) completed the full year of planned therapy. Cytopenias (40.8%) and gastrointestinal adverse events (34.7%) were the most common toxicities. At 1-year post-SCT, overall survival (OS) and relapse-free survival (RFS) were 70% and 67% respectively. Our experience demonstrates single agent ven is a safe, tolerable, and feasible maintenance therapy that may improve RFS and OS in high relapse risk post-SCT patients.

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Fig. 1: Individual patient disease characteristics.
Fig. 2: Individual patient outcomes.
Fig. 3: Transplant outcomes.

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Data availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.

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Acknowledgements

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

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Authors and Affiliations

  1. The University of Colorado School of Medicine, Division of Hematology, Aurora, CO, USA

    Andrew Kent, Marc Schwartz, Christine McMahon, Maria Amaya, Clayton A. Smith, Jennifer Tobin, Kelsey Marciano, Rebecca Rezac, Grace Bosma, Daniel A. Pollyea & Jonathan A. Gutman

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JAG devised this study, supervised implementation of the research, analyzed the data and edited the manuscript. AK analyzed the data and wrote the manuscript. DAP, MS, CM, BB, and CS provided critical input to the analysis and edited the manuscript.

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Correspondence to Jonathan A. Gutman.

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DAP has served as a consultant or advisory board member for Abbvie and Genentech and receives research funding from Abbvie. The other authors declare no competing interests.

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Kent, A., Schwartz, M., McMahon, C. et al. Venetoclax is safe and tolerable as post-transplant maintenance therapy for AML patients at high risk for relapse. Bone Marrow Transplant 58, 849–854 (2023). https://doi.org/10.1038/s41409-023-01987-5

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