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

Chua, CC; Hammond, D; Kent, A; Tiong, IS; Konopleva, MY; Pollyea, DA; DiNardo, CD; Wei, AH

Author(s): Chua, CC; Hammond, D; Kent, A; Tiong, IS; Konopleva, MY; Pollyea, DA; DiNardo, CD; Wei, AH;
Details: Publication Year 2022-07-12,Volume 6,Issue #13,Page 3879-3883
Journal Title: Blood Advances
Publication Type: Research article
Abstract: 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.
Keywords: Aged; *Antineoplastic Combined Chemotherapy Protocols/adverse effects; Bridged Bicyclo Compounds, Heterocyclic/therapeutic use; Humans; *Leukemia, Myeloid, Acute/genetics; Retrospective Studies; Sulfonamides
Department(s): Pathology; Clinical Haematology
PubMed ID: 35511730
Publisher's Version: https://doi.org/10.1182/bloodadvances.2022007083
Open Access at Publisher's Site: https://doi.org/10.1182/bloodadvances.2022007083
Terms of Use/Rights Notice: Refer to copyright notice on published article.

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