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

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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