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

M. S. Thakar, P. Kurre, R. Storb, M. Kletzel, H. Frangoul, M. A. Pulsipher, W. Leisenring, M. E.D. Flowers, B. M. Sandmaier, A. Woolfrey, H. P. Kiem

Research output: Contribution to journalArticlepeer-review

16 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)539-544
Number of pages6
JournalBone marrow transplantation
Volume46
Issue number4
DOIs
StatePublished - Apr 2011

Keywords

  • Fanconi anemia
  • HLA-matched unrelated donor transplant
  • fludarabine

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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