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Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation.

Thakar MS, Kurre P, Storb R, Kletzel M, Frangoul H, Pulsipher MA, Leisenring W, Flowers ME, Sandmaier BM, Woolfrey A, Kiem HP
Bone Marrow Transplant. 2011 46 (4): 539-44

PMID: 20581880 · PMCID: PMC2976796 · DOI:10.1038/bmt.2010.154

A nonmyeloablative conditioning regimen consisting of fludarabine (FLU) and 2 Gy TBI has been used extensively and with substantial engraftment success without promoting excessive nonrelapse mortality in medically infirm patients requiring hematopoietic cell transplantation. In this paper, we studied this same low-toxicity regimen as a means of promoting engraftment of unrelated donor hematopoietic cell transplantation in patients with Fanconi anemia (FA). All patients tolerated the regimen well with no mucositis or other severe toxicities. Of six patients transplanted, five achieved stable mixed or full donor chimerism. Acute and chronic GVHD occurred in four and three patients, respectively. Three patients are alive and well at a median of 45.9 (range, 20.9-68.1) months after transplant. In summary, this FLU-based regimen facilitates stable engraftment of unrelated PBSCs, but is associated with significant chronic GVHD.

MeSH Terms (14)

Child Fanconi Anemia Female Graft vs Host Disease Hematopoietic Stem Cell Transplantation Humans Male Survival Rate Tissue Donors Transplantation Chimera Transplantation Conditioning Treatment Outcome Vidarabine Whole-Body Irradiation

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