HMA/VEN treatment modifications and associated outcomes in IDH-mutant AML Journal Article


Authors: Chin, K. K.; Derkach, A.; Famulare, C.; Gupta, G. K.; Borge, P. D.; Geyer, M.; Goldberg, A. D.; Haque, T.; Park, J. H.; Roeker, L. E.; Tallman, M.; Stahl, M.; Stein, E. M.
Article Title: HMA/VEN treatment modifications and associated outcomes in IDH-mutant AML
Abstract: Hypomethylating agents (HMA) and venetoclax (VEN) are commonly used in patients with IDH-mutated (IDHm) acute myeloid leukemia (AML) ineligible for induction chemotherapy. While prior studies demonstrated high response and survival rates with HMA/VEN in IDHm AML, the impact of treatment modifications in real-world settings is unclear. We retrospectively reviewed 89 IDHm AML patients treated with HMA/VEN from January 2018 to June 2023. CR/CRi rates were 76% in newly diagnosed (ND) and 55% in relapsed/refractory (R/R) patients, and median overall survival was 29.2 months (ND) and 17.1 months (R/R), respectively. Treatment modifications were common. Early VEN reductions were associated with lower response rates but not worse survival. Prolonged cycles were not associated with worse response rates or survival. Significant neutropenia and ED visits or unplanned hospitalizations were considerable before and after CR/CRi, though febrile neutropenia decreased afterward. HMA/VEN is efficacious, with treatment modifications not affecting survival, though long-term toxicities are notable. © 2024 Informa UK Limited, trading as Taylor & Francis Group.
Keywords: controlled study; treatment response; aged; survival rate; major clinical study; overall survival; allogeneic stem cell transplantation; flow cytometry; cancer diagnosis; cohort analysis; retrospective study; hospitalization; immunophenotyping; azacitidine; induction chemotherapy; intensive chemotherapy; demographics; acute myeloid leukemia; decitabine; hypomethylating agent; human; male; female; article; venetoclax; treatment response time; emergency department visit
Journal Title: Leukemia and Lymphoma
Volume: 66
Issue: 2
ISSN: 1042-8194
Publisher: Taylor & Francis Group  
Date Published: 2025-01-01
Start Page: 270
End Page: 278
Language: English
DOI: 10.1080/10428194.2024.2411436
PUBMED: 39397429
PROVIDER: scopus
DOI/URL:
Notes: The MSK Cancer Center Support Grant (P30 CA008748) is acknowledged in PDF. Corresponding MSK author is Eytan M. Stein -- Source: Scopus
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MSK Authors
  1. Eytan Moshe Stein
    363 Stein
  2. Martin Stuart Tallman
    653 Tallman
  3. Jae Hong Park
    373 Park
  4. Mark Blaine Geyer
    89 Geyer
  5. Aaron David Goldberg
    114 Goldberg
  6. Lindsey Elizabeth Roeker
    141 Roeker
  7. Andriy Derkach
    169 Derkach
  8. Gaurav Kumar Gupta
    25 Gupta
  9. Prabhakar Dayanand Borge
    15 Borge
  10. Tamanna Haque
    13 Haque
  11. Kuo-Kai Chin
    9 Chin