Prognostic factors for allogeneic haematopoietic cell transplantation outcomes in primary refractory acute myeloid leukaemia (2013–2022): A retrospective study by the adult acute myeloid leukaemia working group of the Japanese Society for Transplanta
Primary refractory acute myeloid leukaemia (AML) remains a major clinical challenge, with poor outcomes despite salvage chemotherapy. Allogeneic haematopoietic cell transplantation (HCT) continues to be a potentially curative option for these patients. However, recent data on outcomes and prognostic factors specific to primary refractory AML remain limited. We conducted a retrospective analysis of 2600 adult patients with primary refractory AML who underwent their first allogeneic HCT between 2013 and 2022, using data from the Japanese national registry. The 3-year overall survival (OS) and leukaemia-free survival (LFS) rates were 28.5% and 24.4% respectively. The multivariate analysis identified older age (≥50 years), poor performance status (≥2), adverse cytogenetics, extramedullary disease at diagnosis and higher peripheral blood blast count at HCT (≥10%) as significant risk factors for worse OS and LFS. The cumulative incidences of relapse and non-relapse mortality at 3 years were 49.8% and 25.8% respectively. Based on the five significant risk factors, we developed a scoring system that effectively stratified patients into distinct prognostic groups for OS and LFS. This nationwide analysis demonstrated that allogeneic HCT offers the potential for long-term survival in adult patients with primary refractory AML. The proposed risk-scoring system may support clinical decision-making and patient counselling.
Authors
- Masatsugu Tanaka (ORCID: https://orcid.org/0000-0001-8814-230X)
- Naoyuki Uchida (ORCID: https://orcid.org/0000-0001-5952-5926)
- Yoshinobu Kanda (ORCID: https://orcid.org/0000-0002-4866-9307)
- Mamiko Sakata‐Yanagimoto (ORCID: https://orcid.org/0000-0001-7310-8045)
- Masamitsu Yanada (ORCID: https://orcid.org/0000-0003-1602-9775)
- Shohei Mizuno (ORCID: https://orcid.org/0000-0002-4405-8808)
- Yoshiko Atsuta (ORCID: https://orcid.org/0000-0003-4404-2870)
- Toshiro Kawakita (ORCID: https://orcid.org/0000-0001-9595-9529)
- Shuichi Ota (ORCID: https://orcid.org/0000-0002-3631-244X)
- Satoshi Yamasaki (ORCID: https://orcid.org/0000-0002-6143-7906)
- Yoshimitsu Shimomura (ORCID: https://orcid.org/0000-0003-1018-9508)
- Shunsuke Yui
- Junya Kanda (ORCID: https://orcid.org/0000-0002-6704-3633)
- Makoto Onizuka (ORCID: https://orcid.org/0000-0003-3864-0823)
- Noriko Doki
- Satoshi Yoshihara (ORCID: https://orcid.org/0000-0002-8537-2422)
- Masahito Tokunaga (ORCID: https://orcid.org/0000-0003-1296-9006)
- Takahiro Fukuda
- Tetsuya Eto
- Kazuya Ishiwata
- Yuna Katsuoka
- Yuta Katayama
Institutions
- Tokai University (JP)
- Jichi Medical University (JP)
- Aichi Medical University (JP)
- Kanagawa Prefectural Hospital Organization (JP)
- Tokyo Metropolitan Komagome Hospital (JP)
- Nagoya City University (JP)
- St Mary's Hospital (JP)
- Hamanomachi Hospital (JP)
- Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital (JP)
- Sapporo Hokuyu Hospital (JP)
- University of Tsukuba Hospital (JP)
- Sendai Medical Center (JP)
- Imamura Hospital (JP)
- National Hospital Organization (JP)
- Kyoto University Hospital (JP)
- Nippon Medical School Hospital (JP)
- The Japanese Data Center for Hematopoietic Cell Transplantation (JP)
- Kobe City Medical Center General Hospital (JP)
- Kumamoto Medical Center (JP)
- Jichi Medical University Saitama Medical Center (JP)
- Toranomon Hospital (JP)
- Kanagawa Cancer Center
- The University of Tokyo (JP)
- Hyogo University (JP)
Publication Details
- Journal
- British Journal of Haematology
- Published
- 2025-06-16
- DOI
- https://doi.org/10.1111/bjh.20208
- Citations
- 2
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 3.54
Funders
- Japan Agency for Medical Research and Development