TY - JOUR
T1 - Long-term outcome in patients with Fanconi anemia who received hematopoietic stem cell transplantation
T2 - a retrospective nationwide analysis
AU - Yabe, Miharu
AU - Morio, Tomohiro
AU - Tabuchi, Ken
AU - Tomizawa, Daisuke
AU - Hasegawa, Daiichiro
AU - Ishida, Hiroyuki
AU - Yoshida, Nao
AU - Koike, Takashi
AU - Takahashi, Yoshiyuki
AU - Koh, Katsuyoshi
AU - Okamoto, Yasuhiro
AU - Sano, Hideki
AU - Kato, Keisuke
AU - Kanda, Yoshinobu
AU - Goto, Hiroaki
AU - Takita, Junko
AU - Miyamura, Takako
AU - Noguchi, Maiko
AU - Kato, Koji
AU - Hashii, Yoshiko
AU - Astuta, Yoshiko
AU - Yabe, Hiromasa
N1 - Funding Information:
We thank all the clinicians and hospital administrators who provided precise data via the registry of the Japan Society for Stem Cell Transplantation.
Publisher Copyright:
© 2020, Japanese Society of Hematology.
PY - 2021/1
Y1 - 2021/1
N2 - We retrospectively analyzed nationwide records of 163 Fanconi anemia (FA) patients [aplastic anemia (AA), n = 118; myelodysplastic syndrome (MDS), n = 30; acute leukemia, n = 15] who underwent first allogeneic hematopoietic stem cell transplantation (HSCT) between 1987 and 2015 in Japan. An alternative donor was used in 119 (73%) patients, and 160 (98%) patients received a non-T-cell-depleted graft. With an 8.7-year median follow-up, 5-year overall survival (OS) was 81%. The 5-year OS was significantly higher in AA patients than in MDS and acute leukemia patients (89%, 71%, and 44%, respectively). In the MDS/leukemia group, factors associated with poor outcome in univariate analysis were older age at HSCT (≥ 18 years), conditioning regimen without anti-thymocyte or lymphocyte globulin, and grade II–IV acute graft-versus-host disease. After 1 year, of 137 survivors, 15 developed subsequent malignancies, of whom 12 were diagnosed with head and neck (HN)/esophageal cancer. An irradiation regimen and older age were associated with the risk of HN/esophageal cancer. Five of seven deaths were attributed to subsequent malignancies more than 5 years after HSCT. On the basis of the risk factors for HSCT in MDS/leukemia patients and subsequent malignancies, a more effective HSCT approach is required.
AB - We retrospectively analyzed nationwide records of 163 Fanconi anemia (FA) patients [aplastic anemia (AA), n = 118; myelodysplastic syndrome (MDS), n = 30; acute leukemia, n = 15] who underwent first allogeneic hematopoietic stem cell transplantation (HSCT) between 1987 and 2015 in Japan. An alternative donor was used in 119 (73%) patients, and 160 (98%) patients received a non-T-cell-depleted graft. With an 8.7-year median follow-up, 5-year overall survival (OS) was 81%. The 5-year OS was significantly higher in AA patients than in MDS and acute leukemia patients (89%, 71%, and 44%, respectively). In the MDS/leukemia group, factors associated with poor outcome in univariate analysis were older age at HSCT (≥ 18 years), conditioning regimen without anti-thymocyte or lymphocyte globulin, and grade II–IV acute graft-versus-host disease. After 1 year, of 137 survivors, 15 developed subsequent malignancies, of whom 12 were diagnosed with head and neck (HN)/esophageal cancer. An irradiation regimen and older age were associated with the risk of HN/esophageal cancer. Five of seven deaths were attributed to subsequent malignancies more than 5 years after HSCT. On the basis of the risk factors for HSCT in MDS/leukemia patients and subsequent malignancies, a more effective HSCT approach is required.
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U2 - 10.1007/s12185-020-02991-x
DO - 10.1007/s12185-020-02991-x
M3 - Article
C2 - 32949371
AN - SCOPUS:85091062379
SN - 0925-5710
VL - 113
SP - 134
EP - 144
JO - International Journal of Hematology
JF - International Journal of Hematology
IS - 1
ER -
