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

Biol Blood Marrow Transplant. 2007 Dec;13(12):1422-6. doi: 10.1016/j.bbmt.2007.08.050.

Abstract

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 was studied on the patient with ESRD and 2 additional patients with normal renal function. Both patients with SCD achieved full donor erythroid chimerism, have normal blood counts, and are on no immunosuppressive medications. With a 20% dose reduction followed by daily dialysis, we achieved fludarabine drug exposure that is nearly identical to that achieved in patients with normal renal function. We conclude that fludarabine-based nonmyeloablative allogeneic SCT for adult patients with SCD is feasible, even in the setting of ESRD.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural

MeSH terms

  • Adult
  • Anemia, Sickle Cell / complications
  • Anemia, Sickle Cell / therapy*
  • Directed Tissue Donation
  • Female
  • Graft Survival
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Kidney Failure, Chronic / complications*
  • Male
  • Myeloablative Agonists / pharmacokinetics*
  • Myeloablative Agonists / therapeutic use
  • Siblings
  • Transplantation Chimera*
  • Transplantation Conditioning / methods
  • Transplantation, Homologous / methods
  • Vidarabine / analogs & derivatives*
  • Vidarabine / pharmacokinetics
  • Vidarabine / therapeutic use

Substances

  • Myeloablative Agonists
  • Vidarabine
  • fludarabine