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

Monica S. Thakar | Zendy; Peter Kurre | Zendy; R Storb | Zendy; Morris Kletzel | Zendy; Haydar Frangoul | Zendy; Michael A. Pulsipher | Zendy; Wendy M. Leisenring | Zendy; Mary E.D. Flowers | Zendy; Brenda M. Sandmaier | Zendy; Ann E. Woolfrey | Zendy; HP Kiem | Zendy

Open Access

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

Author(s) -

Monica S. Thakar,

Peter Kurre,

R Storb,

Morris Kletzel,

Haydar Frangoul,

Michael A. Pulsipher,

Wendy M. Leisenring,

Mary E.D. Flowers,

Brenda M. Sandmaier,

Ann E. Woolfrey,

HP Kiem

Publication year - 2010

Publication title -

bone marrow transplantation

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.609

H-Index - 127

eISSN - 1476-5365

pISSN - 0268-3369

DOI - 10.1038/bmt.2010.154

Subject(s) - medicine , fanconi anemia , fludarabine , savior sibling , hematopoietic cell , hematopoietic stem cell transplantation , transplantation , anemia , oncology , haematopoiesis , stem cell , chemotherapy , dna repair , cyclophosphamide , genetics , biology , gene

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.

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