Date of Graduation
2001
Document Type
Thesis
Degree Type
MS
Committee Chair
Timothy Tracy
Committee Chair
Timothy Tracy
Abstract
Nephropathic cystinosis is a rare autosomal recessive disorder. Cystinosis results from accumulation of the disulfide amino acid, cystine, within the lysosomes of various cells of the body. Cysteamine bitartrate capsules appear to be an effective and well-tolerated cystine depleting agent and as such have become the standard of therapy for patients with cystinosis. However, the pharmacokinetic parameters of cysteamine bitartrate under steady state conditions have not been fully characterized in the target population as well as the pharmacodynamic relationship to the drug's pharmacokinetic profile. The objectives of this study were to evaluate the pharmacokinetic profile, apparent oral clearance and apparent volume of distribution of cysteamine bitartrate under clinical treatment conditions and to investigate the pharmacokinetic and pharmacodynamic relationship for cysteamine bitartrate under these same conditions. Study results suggest strongly the existence of a pharmacokinetic and pharmacodynamic relationship for cysteamine with respect to its ability to reduce cystine concentrations.
Recommended Citation
Belldina, Eric Bardine, "The treatment of cystinosis with cysteamine." (2001). Graduate Theses, Dissertations, and Problem Reports (ETD). 10596.
https://researchrepository.wvu.edu/etd/10596