Allogeneic haematopoietic cell transplantation from alternative donors with a conditioning regimen of low-dose irradiation, fludarabine and cyclophosphamide in Fanconi anaemia.
A pilot study was undertaken using a fludarabine-based conditioning regimen to improve haematopoietic cell transplantation (HCT) from alternative donors in 27 Fanconi anaemia (FA) patients. Patients were conditioned with 150-180 mg/m2 of fludarabine, 40 mg/kg of cyclophosphamide, 5-10 mg/kg of antithymocyte globulin, and 300-450 cGy of thoracoabdominal/total body irradiation. One patient who received unrelated cord blood transplantation failed to engraft, another patient died of sepsis. The 1-year overall survival was 96.3% (95% CI, 89-100). This conditioning regimen exerted an immunosuppressive effect that enabled durable engraftment in alternative donor HCT without severe toxicity.
Yabe
Hiromasa
H
Specialized Clinical Science, Pediatrics, Tokai University School of Medicine, Isehara, Japan. yabeh@is.icc.u-tokai.ac.jp
Inoue
Hiroyasu
H
Matsumoto
Masae
M
Hamanoue
Satoshi
S
Koike
Takashi
T
Ishiguro
Hiroyuki
H
Koike
Hideki
H
Suzuki
Kazuo
K
Kato
Shunichi
S
Kojima
Seiji
S
Tsuchida
Masahiro
M
Mori
Tetsuya
T
Adachi
Souichi
S
Tsuji
Koichiro
K
Koike
Kenichi
K
Morimoto
Akira
A
Sako
Masahiro
M
Yabe
Miharu
M
D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005199:Fanconi Anemia / Q000532:radiotherapy / Q000628:therapy*; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D008297:Male; D010865:Pilot Projects; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use; D014916:Whole-Body Irradiation
D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine