Gilteritinib versus chemotherapy in Japanese patients with FLT3-mutated relapsed/refractory acute myeloid leukemia

Int J Clin Oncol. 2021 Nov;26(11):2131-2141. doi: 10.1007/s10147-021-02006-7. Epub 2021 Aug 7.


Background: Until recently, no effective targeted therapies for FLT3-mutated (FLT3mut+) relapsed/refractory (R/R) acute myeloid leukemia (AML) were available in Japan. The FLT3 inhibitor, gilteritinib, was approved in Japan for patients with FLT3mut+ R/R AML based on the phase 3 ADMIRAL trial, which demonstrated the superiority of gilteritinib over salvage chemotherapy (SC) with respect to overall survival (OS; median OS, 9.3 vs 5.6 months, respectively; hazard ratio, 0.64 [95% confidence interval 0.49, 0.83]; P < 0.001).

Methods: We evaluated the Japanese subgroup (n = 48) of the ADMIRAL trial, which included 33 patients randomized to 120-mg/day gilteritinib and 15 randomized to SC.

Results: Median OS was 14.3 months in the gilteritinib arm and 9.6 months in the SC arm. The complete remission/complete remission with partial hematologic recovery rate was higher in the gilteritinib arm (48.5%) than in the SC arm (13.3%). After adjustment for drug exposure, fewer adverse events (AEs) occurred in the gilteritinib arm than in the SC arm. Common grade ≥ 3 AEs related to gilteritinib were febrile neutropenia (36%), decreased platelet count (27%), and anemia (24%).

Conclusion: Findings in Japanese patients are consistent with those of the overall ADMIRAL study population.

Keywords: Acute myeloid leukemia; FLT3 inhibitor; FLT3 mutations.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Aniline Compounds
  • Humans
  • Japan
  • Leukemia, Myeloid, Acute* / drug therapy
  • Leukemia, Myeloid, Acute* / genetics
  • Mutation
  • Pyrazines*
  • fms-Like Tyrosine Kinase 3 / genetics


  • Aniline Compounds
  • Pyrazines
  • gilteritinib
  • FLT3 protein, human
  • fms-Like Tyrosine Kinase 3