title: Reduced toxicity and prompt engraftment after minimal conditioning of a patient with Fanconi anemia undergoing hematopoietic stem cell transplantation from an HLA-matched unrelated donor.
abstract: Given the profound sensitivity of patients with Fanconi anemia to conventional conditioning regimens before hematopoietic stem cell transplantation (HSCT), we developed a minimally toxic regimen consisting of 2 Gy total body irradiation, 90 mg/m2 fludarabine, and postgrafting immunosuppression with cyclosporine and mycophenolate to treat FA patients undergoing HSCT from HLA-matched unrelated donors. With over 10 months follow-up, our first patient has complete and sustained engraftment. Graft-versus-host disease was limited to mild skin and liver and moderate gut manifestations. We conclude that the approach is well tolerated and ideally suited to reduce regimen-related toxicities while achieving sustained engraftment.
authors.affiliation: Fred Hutchinson Cancer Research Center and the Department of Pediatrics, University of Washington, Seattle, Washington, U.S.A. pkurre@fhcrc.org
authors.forename: Peter
authors.initials: P
authors.lastname: Kurre
authors.forename: Michael
authors.initials: M
authors.lastname: Pulsipher
authors.forename: Ann
authors.initials: A
authors.lastname: Woolfrey
authors.forename: Michael
authors.initials: M
authors.lastname: Maris
authors.forename: Brenda
authors.initials: B
authors.lastname: Sandmaier
authors.forename: Hans-Peter
authors.initials: HP
authors.lastname: Kiem
authors.forename: Rainier
authors.initials: R
authors.lastname: Storb
mesh_terms: D000328:Adult; D002648:Child; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D006085:Graft Survival; D006650:Histocompatibility Testing; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods; D019520:Living Donors; D008297:Male; D033581:Stem Cell Transplantation / Q000009:adverse effects; D016896:Treatment Outcome; D014916:Whole-Body Irradiation