title: Fludarabine, cyclophosphamide, anti-thymocyteglobulin, and low-dose total body irradiation conditioning enables 1-HLA-locus-mismatched hematopoietic stem cell transplantation for very severe aplastic anemia without affecting ovarian function.
abstract: Allogeneic hematopoietic stem cell transplantation for severe aplastic anemia from an alternative donor is associated with higher risks of graft rejection and severe graft-versus-host disease. We developed a conditioning regimen consisting of rabbit anti-thymocyte globulin, fludarabine, cyclophosphamide, and low-dose total body irradiation. Two adult female patients with transfusion-dependent very severe aplastic anemia underwent 1-locus mismatched transplantation using this regimen. Both patients achieved stable engraftment and the clinical course thereafter was uneventful with persistently normal ovarian function. This novel conditioning regimen may be suitable for alternative donor transplantation for severe aplastic anemia, especially in young female patients.
authors.affiliation: Division of Hematology, Saitama Medical Center, Jichi Medical University, Saitama, Japan.
authors.forename: Shinya
authors.initials: S
authors.lastname: Okuda
authors.forename: Kiriko
authors.initials: K
authors.lastname: Terasako
authors.forename: Kumi
authors.initials: K
authors.lastname: Oshima
authors.forename: Miki
authors.initials: M
authors.lastname: Sato
authors.forename: Hideki
authors.initials: H
authors.lastname: Nakasone
authors.forename: Shinichi
authors.initials: S
authors.lastname: Kako
authors.forename: Rie
authors.initials: R
authors.lastname: Yamazaki
authors.forename: Yukie
authors.initials: Y
authors.lastname: Tanaka
authors.forename: Aki
authors.initials: A
authors.lastname: Tanihara
authors.forename: Takakazu
authors.initials: T
authors.lastname: Higuchi
authors.forename: Junji
authors.initials: J
authors.lastname: Nishida
authors.forename: Yoshinobu
authors.initials: Y
authors.lastname: Kanda
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D003520:Cyclophosphamide / Q000627:therapeutic use; D005260:Female; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D019653:Myeloablative Agonists / Q000627:therapeutic use; D010053:Ovary / Q000187:drug effects / Q000528:radiation effects; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use; D014916:Whole-Body Irradiation / Q000379:methods*
chemical_list: D000961:Antilymphocyte Serum; D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine