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

Publication ,  Journal Article
Horwitz, ME; Spasojevic, I; Morris, A; Telen, M; Essell, J; Gasparetto, C; Sullivan, K; Long, G; Chute, J; Chao, N; Rizzieri, D
Published in: Biol Blood Marrow Transplant
December 2007

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.

Duke Scholars

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Published In

Biol Blood Marrow Transplant

DOI

ISSN

1083-8791

Publication Date

December 2007

Volume

13

Issue

12

Start / End Page

1422 / 1426

Location

United States

Related Subject Headings

  • Vidarabine
  • Transplantation, Homologous
  • Transplantation Conditioning
  • Transplantation Chimera
  • Siblings
  • Myeloablative Agonists
  • Male
  • Kidney Failure, Chronic
  • Immunology
  • Humans
 

Citation

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Horwitz, M. E., Spasojevic, I., Morris, A., Telen, M., Essell, J., Gasparetto, C., … Rizzieri, D. (2007). Fludarabine-based nonmyeloablative stem cell transplantation for sickle cell disease with and without renal failure: clinical outcome and pharmacokinetics. Biol Blood Marrow Transplant, 13(12), 1422–1426. https://doi.org/10.1016/j.bbmt.2007.08.050
Horwitz, Mitchell E., Ivan Spasojevic, Ashley Morris, Marilyn Telen, James Essell, Cristina Gasparetto, Keith Sullivan, et al. “Fludarabine-based nonmyeloablative stem cell transplantation for sickle cell disease with and without renal failure: clinical outcome and pharmacokinetics.Biol Blood Marrow Transplant 13, no. 12 (December 2007): 1422–26. https://doi.org/10.1016/j.bbmt.2007.08.050.
Horwitz ME, Spasojevic I, Morris A, Telen M, Essell J, Gasparetto C, et al. 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.
Horwitz, Mitchell E., et al. “Fludarabine-based nonmyeloablative stem cell transplantation for sickle cell disease with and without renal failure: clinical outcome and pharmacokinetics.Biol Blood Marrow Transplant, vol. 13, no. 12, Dec. 2007, pp. 1422–26. Pubmed, doi:10.1016/j.bbmt.2007.08.050.
Horwitz ME, Spasojevic I, Morris A, Telen M, Essell J, Gasparetto C, Sullivan K, Long G, Chute J, Chao N, Rizzieri D. 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):1422–1426.
Journal cover image

Published In

Biol Blood Marrow Transplant

DOI

ISSN

1083-8791

Publication Date

December 2007

Volume

13

Issue

12

Start / End Page

1422 / 1426

Location

United States

Related Subject Headings

  • Vidarabine
  • Transplantation, Homologous
  • Transplantation Conditioning
  • Transplantation Chimera
  • Siblings
  • Myeloablative Agonists
  • Male
  • Kidney Failure, Chronic
  • Immunology
  • Humans