Treatment-free remission after ceasing venetoclax-based therapy in patients with acute myeloid leukemia

Blood Adv. 2022 Jul 12;6(13):3879-3883. doi: 10.1182/bloodadvances.2022007083.


The clinical benefit of adding venetoclax (VEN) to hypomethylating agents or low-dose cytarabine in older and/or unfit patients with newly diagnosed acute myeloid leukemia (AML) has been confirmed in phase 3 studies. With the increased uptake of VEN-based therapies for patients with AML, a pertinent question is whether treatment can be safely ceased among patients who have achieved sustained remission. We hypothesized that a proportion of patients opting to cease therapy may benefit from a treatment-free remission (TFR) period without indefinite treatment. We report the retrospective outcomes of 29 patients in remission for a minimum of 12 months on VEN-based therapy, with 55% continuing therapy until disease progression and 45% electively ceasing treatment (STOP). With follow-up exceeding 5 years, we observed a median TFR lasting 45.8 months among the STOP cohort, with >50% of patients still in sustained remission at the data cutoff. The risk of relapse and duration of relapse-free and overall survival were similar between the 2 cohorts. Factors favoring sustained TFR within the cohort included NPM1 and/or IDH2 mutation at diagnosis, complete remission without measurable residual disease, and at least 12 months of VEN-based combination therapy prior to treatment cessation.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Antineoplastic Combined Chemotherapy Protocols* / adverse effects
  • Bridged Bicyclo Compounds, Heterocyclic / therapeutic use
  • Humans
  • Leukemia, Myeloid, Acute* / genetics
  • Retrospective Studies
  • Sulfonamides


  • Bridged Bicyclo Compounds, Heterocyclic
  • Sulfonamides
  • venetoclax