title: Allogeneic hematopoietic stem cell transplantation of patients with FA and high risk features using fludarabine without radiation.
abstract: 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.
authors.affiliation: Division of Hematology-Oncology, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
authors.forename: Jillian M
authors.initials: JM
authors.lastname: Baker
authors.forename: Victor A
authors.initials: VA
authors.lastname: Lewis
authors.forename: Conrad V
authors.initials: CV
authors.lastname: Fernandez
authors.forename: Michel
authors.initials: M
authors.lastname: Duval
authors.forename: Bruce N
authors.initials: BN
authors.lastname: Crooks
authors.forename: Kim
authors.initials: K
authors.lastname: Yuille
authors.forename: Melvin H
authors.initials: MH
authors.lastname: Freedman
authors.forename: John J
authors.initials: JJ
authors.lastname: Doyle
authors.forename: Yigal
authors.initials: Y
authors.lastname: Dror
mesh_terms: 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
chemical_list: D014740:Vidarabine; C024352:fludarabine