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.
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.
Okuda
Shinya
S
Division of Hematology, Saitama Medical Center, Jichi Medical University, Saitama, Japan.
Terasako
Kiriko
K
Oshima
Kumi
K
Sato
Miki
M
Nakasone
Hideki
H
Kako
Shinichi
S
Yamazaki
Rie
R
Tanaka
Yukie
Y
Tanihara
Aki
A
Higuchi
Takakazu
T
Nishida
Junji
J
Kanda
Yoshinobu
Y
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*
D000961:Antilymphocyte Serum; D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine