{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Konuma T"],"funding":["AMED","Japan Agency for Medical Research and Development"],"pagination":["484-497"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12379025"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["207(2)"],"pubmed_abstract":["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."],"journal":["British journal of haematology"],"pubmed_title":["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 Transplantation and Cellular Therapy."],"pmcid":["PMC12379025"],"funding_grant_id":["18ek0510023h0002"],"pubmed_authors":["Doki N","Ota S","Shimomura Y","Uchida N","Yamasaki S","Katsuoka Y","Katayama Y","Sakata-Yanagimoto M","Tokunaga M","Kanda J","Fukuda T","Kawakita T","Eto T","Konuma T","Mizuno S","Onizuka M","Ishiwata K","Kanda Y","Atsuta Y","Yanada M","Yui S","Yoshihara S","Tanaka M"],"additional_accession":[]},"is_claimable":false,"name":"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 Transplantation and Cellular Therapy.","description":"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.","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Aug","modification":"2026-05-10T01:51:55.452Z","creation":"2026-05-08T03:04:55.26Z"},"accession":"S-EPMC12379025","cross_references":{"pubmed":["40518826"],"doi":["10.1111/bjh.20208"]}}