Gilteritinib with or without venetoclax for relapsed/refractory FLT3-mutated acute myeloid leukaemia

Eitan Kugler, Inbar Cohen, Irina Amitai, Ron Ram, Avraham Frisch, Boaz Nachmias, Jonathan Canaani, Yakir Moshe, Baher Krayem, Shlomzion Aumann, Israel Henig, Vladimir Vainstein, Liat Shargian, Chezi Ganzel, Moshe Yeshurun, Itay Levi, Pia Raanani, Luiza Akria, Yishai Ofran, Shai ShimonyOfir Wolach

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Patients with FLT3-mutated acute myeloid leukaemia (AML) that relapse or are refractory (R/R) to intensive induction have poor outcomes. Gilteritinib has recently become standard-of-care for patients with R/R FLT3-mutated AML. We investigated whether adding venetoclax to gilteritinib (gilt-ven) improves outcomes as compared with gilteritinib monotherapy. We included patients treated with gilteritinib (n = 19) and gilt-ven (n = 17) for R/R AML after intensive chemotherapy. Gilteritinib and gilt-ven groups did not differ in terms of mCRc rates (53% and 65%, p = 0.51) and realization of allogeneic haematopoietic stem-cell transplantation (HSCT, 47% and 35%, p = 0.5). Overall survival (OS) was comparable between groups, although a trend towards better OS was seen with gilt-ven (12-month OS 58.8% [95% CI 39.5%–87.6%]) versus gilteritinib (42.1% [95% CI 24.9%–71.3%] for gilteritinib). Early salvage with gilt-ven versus any other gilteritinib-based approach was associated with the best outcome (p = 0.031). Combination therapy was associated with increased haematological toxicity. In summary, gilt-ven did not improve remissions or HSCT-realization rates in patients with R/R FLT3-mutated AML as compared with gilteritinib and was associated with increased haematological toxicity. Although OS did not differ, a trend towards better survival was suggested with gilt-ven and a survival benefit was shown for gilt-ven approach when sequenced early for salvage.

Original languageEnglish
JournalBritish Journal of Haematology
Early online date23 May 2024
StateE-pub ahead of print - 23 May 2024

Bibliographical note

Publisher Copyright:
© 2024 British Society for Haematology and John Wiley & Sons Ltd.


  • acute myeloid leukaemia (AML)
  • Fms-related receptor tyrosine kinase 3 (FLT3) mutation
  • gilteritinib
  • venetoclax


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