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Successful engraftment without radiation after fludarabine‐based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation
Author(s) -
Tan PohLin,
Wagner John E.,
Auerbach Arleen D.,
DeFor Todd E.,
Slungaard Arne,
MacMillan Margaret L.
Publication year - 2005
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.20538
Subject(s) - medicine , fludarabine , transplantation , gastroenterology , hematopoietic stem cell transplantation , fanconi anemia , total body irradiation , aplastic anemia , surgery , regimen , cyclophosphamide , bone marrow , chemotherapy , biochemistry , chemistry , dna repair , gene
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. © 2005 Wiley‐Liss, Inc.