Gilteritinib monotherapy for relapsed/refractory FLT3 mutated acute myeloid leukemia: a real-world, multi-center, matched analysis

Shai Shimony*, Jonathan Canaani, Eitan Kugler, Boaz Nachmias, Ron Ram, Israel Henig, Avraham Frisch, Chezi Ganzel, Vladimir Vainstein, Yakir Moshe, Shlomzion Aumann, Moshe Yeshurun, Yishai Ofran, Pia Raanani, Ofir wolach

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


Patients with FLT3-mutated relapsed or refractory (R/R) acute myeloid leukemia (AML) have a dismal prognosis. Gilteritinib is a FLT3 tyrosine kinase inhibitor (TKI) recently approved for patients with R/R AML. We aimed to characterize real-world data regarding gilteritinib treatment in FLT3-mutated R/R AML and to compare outcomes with matched FLT3-mutated R/R AML patients treated with chemotherapy-based salvage regimens. Twenty-five patients from six academic centers were treated with gilteritinib for FLT3-mutated R/R AML. Eighty percent were treated with a prior intensive induction regimen and 40% of them received prior TKI therapy. Twelve patients (48%) achieved complete response (CR) with gilteritinib. The estimated median overall survival (OS) of the entire cohort was eight (CI 95% 0–16.2) months and was significantly higher in patients who achieved CR compared to those who did not (16.3 months, CI 95% 0–36.2 vs. 2.6 months, CI 95% 1.47–3.7; p value = 0.046). In a multivariate cox regression analysis, achievement of CR was the only predictor for longer OS (HR 0.33 95% CI 0.11–0.97, p = 0.044). Prior TKI exposure did not affect OS but was associated with better event-free survival (HR 0.15 95% CI 0.03–0.71, p = 0.016). An age and ELN-risk matched comparison between patients treated with gilteritinib and intensive salvage revealed similar response rates (50% in both groups); median OS was 9.6 months (CI 95% 2.3–16.8) vs. 7 months (CI 95% 5.1–8.9) in gilteritinib and matched controls, respectively (p = 0.869). In conclusion, in the real-world setting, gilteritinib is effective, including in heavily pre-treated, TKI exposed patients.

Original languageEnglish
Pages (from-to)2001-2010
Number of pages10
JournalAnnals of Hematology
Issue number9
StatePublished - Sep 2022


  • FLT3
  • Gilteritinib
  • Relapsed/refractory AML


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