Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease

K. van Besien*, A. Bartholomew, W. Stock, D. Peace, S. Devine, D. Sher, J. Sosman, Y. H. Chen, M. Koshy, R. Hoffman

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

83 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)445-449
Number of pages5
JournalBone Marrow Transplantation
Issue number4
StatePublished - 2000


  • Allogeneic transplantation
  • Fludarabine
  • Renal failure
  • Sickle cell disease

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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