Long-term outcome in patients with Fanconi anemia who received hematopoietic stem cell transplantation: a retrospective nationwide analysis.
Acute Disease
Adolescent
Adult
Age Factors
Child
Child, Preschool
Esophageal Neoplasms
/ etiology
Fanconi Anemia
/ complications
Female
Follow-Up Studies
Head and Neck Neoplasms
/ etiology
Hematopoietic Stem Cell Transplantation
/ adverse effects
Humans
Infant
Japan
Leukemia
/ etiology
Male
Middle Aged
Myelodysplastic Syndromes
/ etiology
Retrospective Studies
Survival Rate
Time Factors
Transplantation, Homologous
Treatment Outcome
Young Adult
Acute leukemia
Fanconi anemia
Hematopoietic stem cell transplantation
Myelodysplastic syndrome
Subsequent malignancies after hematopoietic stem cell transplantation
Journal
International journal of hematology
ISSN: 1865-3774
Titre abrégé: Int J Hematol
Pays: Japan
ID NLM: 9111627
Informations de publication
Date de publication:
Jan 2021
Jan 2021
Historique:
received:
25
05
2020
accepted:
28
08
2020
revised:
22
08
2020
pubmed:
20
9
2020
medline:
12
2
2021
entrez:
19
9
2020
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 32949371
doi: 10.1007/s12185-020-02991-x
pii: 10.1007/s12185-020-02991-x
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
134-144Références
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