Fludarabine-based nonmyeloablative stem cell transplantation for sickle cell disease with and without renal failure: clinical outcome and pharmacokinetics.

@article{Horwitz2007FludarabinebasedNS,
  title={Fludarabine-based nonmyeloablative stem cell transplantation for sickle cell disease with and without renal failure: clinical outcome and pharmacokinetics.},
  author={Mitchell E Horwitz and Ivan Spasojevi{\'c} and Ashley K Morris and Marilyn J Telen and James H. Essell and Cristina J. Gasparetto and Keith M. Sullivan and Gwynn D. Long and John P. Chute and Nelson J Chao and David Alan Rizzieri},
  journal={Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation},
  year={2007},
  volume={13 12},
  pages={1422-6}
}
End-organ damage is common in patients with sickle cell disease (SCD) thereby limiting the use of allogeneic stem cell transplantation (SCT). We report the outcome of 2 adult SCD patients, 1 with end-stage renal disease (ESRD), who underwent fludarabine-based nonmyeloablative SCT from HLA-identical matched siblings. To prevent fludarabine toxicity, the patient with ESRD underwent aggressive dialysis following adjusted fludarabine dosing. Pharmacokinetics of the fludarabine metabolite F-Ara-A… CONTINUE READING

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