Venetoclax plus azacitidine compared with intensive chemotherapy as induction for patients with acute myeloid leukemia: retrospective analysis of an electronic medical record database in the United States

Ann Hematol. 2023 Apr;102(4):749-754. doi: 10.1007/s00277-023-05109-5. Epub 2023 Feb 3.

Abstract

Intensive chemotherapy (IC) is commonly used to achieve remission in patients with acute myeloid leukemia (AML). Venetoclax plus azacitidine (VEN-AZA) is FDA-approved to treat patients with AML aged ≥ 75 years or who are ineligible for IC. This retrospective analysis used de-identified electronic health records from the US-based Flatiron Health database from patients diagnosed 11/21/2018 to 10/31/2021 to compare treatment outcomes with VEN-AZA vs. IC. Patients were 1:1 propensity score-matched ([Formula: see text]). Assessments included rates of complete remission (CR) and hematopoietic stem cell transplant (HSCT), overall survival (OS), and relapse-free survival (RFS). CR and HSCT rates were higher with IC than with VEN-AZA (60.9% vs. 44.2% [P = 0.006] and 18.1% vs. 8.0% [P = 0.012], respectively). Median OS was 17.7 months in patients treated with IC and 11.3 months with VEN-AZA without censoring (P = 0.278) and 13.7 vs. 10.6 months, respectively, with censoring at HSCT (P = 0.584). Median RFS was 12.0 months in patients treated with IC vs. 9.5 months with VEN-AZA without censoring (P = 0.431) and 6.4 vs. 7.4 months, respectively, with censoring at HSCT (P = 0.444). No OS or RFS differences observed between the two arms reached statistical significance. Randomized controlled trials comparing the two approaches are warranted, as are novel approaches to reduce relapse rates following CR.

Keywords: Acute myeloid leukemia; Clinical outcomes; Intensive chemotherapy; Venetoclax plus azacitidine.

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Azacitidine / therapeutic use
  • Bridged Bicyclo Compounds, Heterocyclic / therapeutic use
  • Electronic Health Records*
  • Humans
  • Leukemia, Myeloid, Acute* / therapy
  • Retrospective Studies
  • United States / epidemiology

Substances

  • venetoclax
  • Azacitidine
  • Bridged Bicyclo Compounds, Heterocyclic