Allogeneic hematopoietic stem cell transplantation of patients with FA and high risk features using fludarabine without radiation.
Several factors unique to Fanconi anemia (FA) limit the success of allogeneic hematopoietic stem cell transplantation (HSCT) in this population. In this report, we describe a multi-center pilot study of five consecutive FA patients with high-risk features for transplant prepared with fludarabine, without radiation. Four patients engrafted quickly, experienced minimal toxicity and are well at 43-65 months post-transplant. One patient had a C-mismatched unrelated donor transplant and had unsustained engraftment. This fludarabine based regimen without radiation was safe and effective for four high-risk patients, suggesting that eliminating radiation should be further studied as an approach to HSCT in children with FA.
Baker
Jillian M
JM
Division of Hematology-Oncology, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
Lewis
Victor A
VA
Fernandez
Conrad V
CV
Duval
Michel
M
Crooks
Bruce N
BN
Yuille
Kim
K
Freedman
Melvin H
MH
Doyle
John J
JJ
Dror
Yigal
Y
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005199:Fanconi Anemia / Q000601:surgery*; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival / Q000187:drug effects; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D010865:Pilot Projects; D011827:Radiation; D012307:Risk Factors; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000494:pharmacology
D014740:Vidarabine; C024352:fludarabine