Fludarabine-based nonmyeloablative stem cell transplantation for sickle cell disease with and without renal failure: clinical outcome and pharmacokinetics.
Journal Article (Journal Article)
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.
Full Text
Duke Authors
- Chao, Nelson Jen An
- Gasparetto, Cristina
- Horwitz, Mitchell Eric
- Long, Gwynn Douglas
- Spasojevic, Ivan
- Sullivan, Keith Michael
- Telen, Marilyn Jo
Cited Authors
- Horwitz, ME; Spasojevic, I; Morris, A; Telen, M; Essell, J; Gasparetto, C; Sullivan, K; Long, G; Chute, J; Chao, N; Rizzieri, D
Published Date
- December 2007
Published In
Volume / Issue
- 13 / 12
Start / End Page
- 1422 - 1426
PubMed ID
- 18022571
Pubmed Central ID
- PMC2153438
International Standard Serial Number (ISSN)
- 1083-8791
Digital Object Identifier (DOI)
- 10.1016/j.bbmt.2007.08.050
Language
- eng
Conference Location
- United States