title: Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation.
abstract: A nonmyeloablative conditioning regimen consisting of fludarabine (FLU) and 2 Gy TBI has been used extensively and with substantial engraftment success without promoting excessive nonrelapse mortality in medically infirm patients requiring hematopoietic cell transplantation. In this paper, we studied this same low-toxicity regimen as a means of promoting engraftment of unrelated donor hematopoietic cell transplantation in patients with Fanconi anemia (FA). All patients tolerated the regimen well with no mucositis or other severe toxicities. Of six patients transplanted, five achieved stable mixed or full donor chimerism. Acute and chronic GVHD occurred in four and three patients, respectively. Three patients are alive and well at a median of 45.9 (range, 20.9-68.1) months after transplant. In summary, this FLU-based regimen facilitates stable engraftment of unrelated PBSCs, but is associated with significant chronic GVHD.
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, WA 19024, USA.
authors.forename: M S
authors.initials: MS
authors.lastname: Thakar
authors.forename: P
authors.initials: P
authors.lastname: Kurre
authors.forename: R
authors.initials: R
authors.lastname: Storb
authors.forename: M
authors.initials: M
authors.lastname: Kletzel
authors.forename: H
authors.initials: H
authors.lastname: Frangoul
authors.forename: M A
authors.initials: MA
authors.lastname: Pulsipher
authors.forename: W
authors.initials: W
authors.lastname: Leisenring
authors.forename: M E D
authors.initials: ME
authors.lastname: Flowers
authors.forename: B M
authors.initials: BM
authors.lastname: Sandmaier
authors.forename: A
authors.initials: A
authors.lastname: Woolfrey
authors.forename: H-P
authors.initials: HP
authors.lastname: Kiem
mesh_terms: D002648:Child; D005199:Fanconi Anemia / Q000188:drug therapy / Q000628:therapy*; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D008297:Male; D015996:Survival Rate; D014019:Tissue Donors; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000009:adverse effects* / Q000379:methods; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000633:toxicity; D014916:Whole-Body Irradiation / Q000009:adverse effects
chemical_list: D014740:Vidarabine; C024352:fludarabine