• Amanda C Winters, Jonathan A Gutman, Enkhtsetseg Purev, Molly Nakic, Jennifer Tobin, Stephanie Chase, Jeff Kaiser, Lindsey Lyle, Chelsey Boggs, Keri Halsema, Jeffrey T Schowinsky, Julie Rosser, Mark D Ewalt, Bradford Siegele, Vishal Rana, Steven Schuster, Diana Abbott, Brett M Stevens, Craig T Jordan, Clayton Smith, and Daniel A Pollyea.
• Center for Cancer and Blood Disorders, Department of Pediatrics, University of Colorado Denver, Aurora, CO.
• Blood Adv. 2019 Oct 22; 3 (20): 2911-2919.

AbstractVenetoclax is approved for older untreated acute myeloid leukemia (AML) patients. Venetoclax was available prior to approval off-label. We assessed our single-institution off-label experience with venetoclax/azacitidine, comparing outcomes with a clinical trial cohort that administered this regimen at the same institution. Thirty-three untreated AML patients unfit or unwilling to receive induction chemotherapy and prescribed venetoclax/azacitidine off-trial were retrospectively analyzed and compared with 33 patients who received the same therapy on trial. Outcomes were compared, and comparisons were made to a theoretical scenario in which off-trial patients received induction. Digital droplet polymerase chain reaction evaluated measurable residual disease (MRD). Off-trial venetoclax was attainable in nearly all patients for whom this was desired. The complete remission (CR)/CR with incomplete blood count recovery rate was 63.3% for off-trial patients who received treatment and 84.9% for trial patients (P = .081). The median overall survival for off-trial patients who received treatment was 381 days (95% confidence interval [CI], 174, not reached) vs 880 days (95% CI, 384, not reached) for trial patients (P = .041). Prior exposure to hypomethylating agents was associated with worse outcomes. Response rates with venetoclax/azacitidine were not inferior to a theoretical scenario in which patients received induction, and early death rates were less than expected with induction. MRD negativity was achievable. Newly diagnosed AML patients treated in a "real-world" scenario with off-trial venetoclax/azacitidine had inferior outcomes compared with patients treated in the setting of a clinical trial. Additionally, this therapy may be as effective, and less toxic, when compared with induction chemotherapy.© 2019 by The American Society of Hematology.

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