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.
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.
Kurre
Peter
P
Fred Hutchinson Cancer Research Center and the Department of Pediatrics, University of Washington, Seattle, Washington, U.S.A. pkurre@fhcrc.org
Pulsipher
Michael
M
Woolfrey
Ann
A
Maris
Michael
M
Sandmaier
Brenda
B
Kiem
Hans-Peter
HP
Storb
Rainier
R
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