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- Title
Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease.
- Authors
van Besien, K; Bartholomew, A; Stock, W; Peace, D; Devine, S; Sher, D; Sosman, J; Chen, Y-H; Koshy, M; Hoffman, R
- Abstract
Although allogeneic transplantation can be curative for patients with sickle cell disease, the toxicity of conditioning regimens has precluded its use in adults with significant end-organ damage. Newer conditioning regimens have been developed that are less toxic and that may broaden the applicability of allogeneic transplantation in this disorder. We report two adults with end-stage sickle cell disease, who underwent allogeneic transplantation from an HLA-identical sibling donor after conditioning with fludarabine/melphalan and ATG. Both patients had been extensively transfused and one had multiple RBC antibodies. One of the patients also had end-stage renal disease, and was dialysis dependent. Engraftment occurred promptly in both patients. Both achieved 100% donor chimerism and both were free of pain crises after transplant. The first patient died of a respiratory failure related to chronic graft-versus-host disease (GVHD) on day 335 after transplantation. The second patient developed severe gastro-intestinal GVHD and TTP and died on day 147 after transplantation. Conditioning with fludarabine/melphalan and ATG followed by allogeneic stem cell transplantation resulted in prompt and reliable engraftment in adults with end-stage sickle cell disease. The incidence of severe GVHD was unacceptably high and may be related to the ethnicity of the patients or to the inflammatory state associated with pre-existing sickle cell disease. Bone Marrow Transplantation (2000) 26, 445–449.
- Subjects
SICKLE cell anemia; GRAFT versus host disease; CELL transplantation
- Publication
Bone Marrow Transplantation, 2000, Vol 26, Issue 4, p445
- ISSN
0268-3369
- Publication type
Article
- DOI
10.1038/sj.bmt.1702518