BACKGROUND: To potentially reduce late effects of malignancy, chronic 
graft-versus-host disease (GVHD), endocrinopathy, and infertility in patients 
with Fanconi anemia (FA) undergoing HLA-matched related donor hematopoietic cell 
transplantation (HCT), we developed a regimen using fludarabine (FLU), 
cyclophosphamide (CY), and anti-thymocyte globulin (ATG) followed by infusion of 
T-cell depleted (TCD) bone marrow (BM) or unmanipulated umbilical cord blood 
(UCB). GVHD prophylaxis consisted of cyclosporine and short course 
methylprednisolone.
PROCEDURE: Between April 2000 and June 2003, 11 patients (10 aplastic anemia 
(AA), 1 myelodysplastic syndrome (MDS)) underwent HCT using this regimen. Stem 
cell sources were BM and UCB in eight and three patients, respectively.
RESULTS: All patients demonstrated primary engraftment. Median days to 
neutrophil and platelet engraftment were 11 days (range 9-21) and 38 days (range 
19-381), respectively. No patient developed GVHD after primary HCT. The patient 
with MDS relapsed with AML and a maternal donor recipient experienced secondary 
graft failure. For the nine FA patients with AA who underwent HLA-identical 
sibling donor HCT, the Kaplan-Meier estimates of overall survival and event-free 
survival (EFS) at 2 years are 100% and 82%, respectively, at a median follow-up 
of 2.9 years (range 1.9-4.8).
CONCLUSIONS: In summary, a FLU-based, non-irradiation approach is effective for 
FA patients with AA undergoing HLA-identical sibling donor HCT.