Maintenance treatment with gilteritinib suppresses post-transplant relapse in relapse/refractory FLT3-mutated acute myeloid leukemia
The FLT3 inhibitor, gilteritinib, has been widely used in patients with relapsed or refractory (R/R) FLT3-mutated acute myeloid leukemia (AML), but its tolerability and effectiveness after allogeneic hematopoietic stem cell transplantation (HSCT) are not well known outside of clinical trials.Therefore, we performed nation-wide surveillance in Japan. We used data from the national registry for patients transplanted including R/R FLT3-mutated AML patients treated with gilteritinib before and/or after HSCT. Post-HSCT administration dose and safety were summarized, and outcomes, including relapse-free survival (RFS) were investigated. We also used the historical cohort of HSCT before the approval of gilteritinib. Among R/R FLT3-mutated AML patients, 120 were treated with gilteritinib before and/or after allo-HSCT, and maintenance treatment was performed in 55 patients. The median initiation day was Day 47 after allo-HSCT (range 23-379) with a median starting dose of 80 mg (range 40-120 mg), and serious adverse events leading to gilteritinib dose reduction or temporary discontinuation were observed in 52.7% of these cases. The 3-year RFS was 46.8% and patients treated with post-HSCT gilteritinib had significantly better RFS (58.8%) than those without it (36.4%) (p < 0.001). Survival data in the historical cohort were similar to that of those who did not resume gilteritinib. In the subgroup analysis, post-HSCT gilteritinib showed an RFS benefit in cord blood transplantation (CBT; 79.4% vs. 26.1%, p < 0.001), but not in bone marrow or peripheral blood stem cell transplantation. This Japanese real-world study supports the tolerability and effectiveness of post-HSCT gilteritinib in R/R FLT3-mutated AML.
Authors
- Masatsugu Tanaka (ORCID: https://orcid.org/0000-0001-8814-230X)
- Akiyoshi Takami (ORCID: https://orcid.org/0000-0002-1822-9976)
- Yoshinobu Kanda (ORCID: https://orcid.org/0000-0002-4866-9307)
- Yuta Katayama (ORCID: https://orcid.org/0000-0001-5054-9104)
- Noboru Asada (ORCID: https://orcid.org/0000-0001-7322-5460)
- Masamitsu Yanada (ORCID: https://orcid.org/0000-0003-1602-9775)
- Mio Kurata (ORCID: https://orcid.org/0009-0002-5065-1672)
- Noriko Doki (ORCID: https://orcid.org/0000-0002-8661-3179)
- Kazunori Imada (ORCID: https://orcid.org/0000-0002-0945-0513)
- Takanori Teshima (ORCID: https://orcid.org/0000-0002-0941-271X)
- Yoshiko Atsuta (ORCID: https://orcid.org/0000-0003-4404-2870)
- Satoshi Uno (ORCID: https://orcid.org/0000-0002-8209-6864)
- Takaaki Konuma (ORCID: https://orcid.org/0000-0002-1953-8463)
- Yasuyuki Arai (ORCID: https://orcid.org/0000-0002-9662-5093)
- Marie Ohbiki (ORCID: https://orcid.org/0009-0007-3522-2278)
- Koichi Onodera
- Mamiko Sakata-Yanagimoto
- Takashi Ishida
- Shingo Yano
- Shuichi Ota
- Takahiro Fukuda
- Nobuhiro Hiramoto
Institutions
- Hokkaido University of Science (JP)
- Jikei University School of Medicine (JP)
- Astellas Pharma (Japan) (JP)
- Jichi Medical University (JP)
- Health Sciences University of Hokkaido (JP)
- Aichi Medical University (JP)
- Hokkaido University (JP)
- Kyoto University (JP)
- Kanagawa Prefectural Hospital Organization (JP)
- Tokyo Metropolitan Komagome Hospital (JP)
- Nagoya City University (JP)
- Tohoku University Hospital (JP)
- Okayama University Hospital (JP)
- Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital (JP)
- Sapporo Hokuyu Hospital (JP)
- University of Tsukuba Hospital (JP)
- Osaka Hospital (JP)
- The Japanese Data Center for Hematopoietic Cell Transplantation (JP)
- Kobe City Medical Center General Hospital (JP)
- Kanagawa Cancer Center
- The University of Tokyo (JP)
Publication Details
- Journal
- Transplantation and Cellular Therapy
- Published
- 2026-04-01
- DOI
- https://doi.org/10.1016/j.jtct.2026.03.042
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Astellas Pharma