title: Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia.
abstract: We developed a haploidentical transplantation protocol with post-transplant cyclophosphamide (CY) for in vivo T-cell depletion (TCD) using a novel adapted-dosing schedule (25 mg/kg on days +3 and +4) for Fanconi anemia (FA). With median follow-up of 3 years (range, 37 days to 6.2 years), all six patients engrafted. Two patients with multiple pre-transplant comorbidities died, one from sepsis and one from sepsis with associated chronic GVHD. Four patients without preexisting comorbidities and early transplant referrals are alive with 100% donor chimerism and excellent performance status. We conclude that adjusted-dosing post-transplant CY is effective in in vivo TCD to promote full donor engraftment in patients with FA.
authors.affiliation: Pediatric Hematology Oncology, Medical College of Wisconsin and Children's Hospital of Wisconsin, Milwaukee, WI, USA.
authors.forename: M S
authors.initials: MS
authors.lastname: Thakar
authors.affiliation: Hospital de Clinicas, Universidade do Parana, Curitiba Brazil.
authors.forename: C
authors.initials: C
authors.lastname: Bonfim
authors.affiliation: UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
authors.forename: M C
authors.initials: MC
authors.lastname: Walters
authors.affiliation: Fred Hutchinson Cancer Research Center and University of Washington, Seattle, WA, USA.
authors.forename: R
authors.initials: R
authors.lastname: Storb
authors.affiliation: Hospital de Clinicas, Universidade do Parana, Curitiba Brazil.
authors.forename: R
authors.initials: R
authors.lastname: Pasquini
authors.affiliation: Fred Hutchinson Cancer Research Center and University of Washington, Seattle, WA, USA.|Seattle Children's Hospital, Seattle, WA, USA.
authors.forename: L
authors.initials: L
authors.lastname: Burroughs
authors.affiliation: Fred Hutchinson Cancer Research Center and University of Washington, Seattle, WA, USA.
authors.forename: B M
authors.initials: BM
authors.lastname: Sandmaier
authors.affiliation: Fred Hutchinson Cancer Research Center and University of Washington, Seattle, WA, USA.|Seattle Children's Hospital, Seattle, WA, USA.
authors.forename: A
authors.initials: A
authors.lastname: Woolfrey
authors.affiliation: Fred Hutchinson Cancer Research Center and University of Washington, Seattle, WA, USA.
authors.forename: H-P
authors.initials: HP
authors.lastname: Kiem
mesh_terms: D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D004334:Drug Administration Schedule; D005199:Fanconi Anemia / Q000401:mortality / Q000628:therapy*; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D008212:Lymphocyte Depletion / Q000379:methods*; D008297:Male; D013601:T-Lymphocytes; D000075442:Transplantation, Haploidentical / Q000379:methods*
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide