The propriety of upgrading responses to venetoclax + azacitidine in newly diagnosed patients with acute myeloid leukemia

Leuk Lymphoma. 2021 Jun;62(6):1466-1473. doi: 10.1080/10428194.2020.1864358. Epub 2020 Dec 30.


Widely-used response criteria, conditional upon count recovery, were developed for acute myeloid leukemia (AML) in the context of intensive chemotherapy (IC). Extending these definitions to continuously-administered venetoclax-based therapies might underestimate responses. Best practices for venetoclax-based therapies mandate interruption after an end-of-cycle 1 bone marrow biopsy shows morphologic remission with cytopenias. We analyzed 435 patients with newly-diagnosed AML and follow-up response assessments. Of the 101 who responded to venetoclax + azacitidine, overall survival for patients whose response was upgraded due to count recovery during a 14-day post-disease assessment period, from complete remission (CR) with incomplete recovery of blood counts to CR, was not different compared to patients who did not need the 14-day period for count recovery. These results were distinct from 138 IC patients. Although sample sizes for the comparison groups were small, and conclusions are exploratory and must be verified, these findings support consideration of new response criteria for venetoclax-based regimens.

Keywords: AML; ELN; response; upgrade; venetoclax.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Azacitidine* / therapeutic use
  • Bridged Bicyclo Compounds, Heterocyclic / therapeutic use
  • Humans
  • Leukemia, Myeloid, Acute* / diagnosis
  • Leukemia, Myeloid, Acute* / drug therapy
  • Sulfonamides


  • Bridged Bicyclo Compounds, Heterocyclic
  • Sulfonamides
  • Azacitidine
  • venetoclax