Pharmacokinetics of cysteamine in a cystinosis patient treated with hemodialysis
- نوع فایل : کتاب
- زبان : انگلیسی
- مؤلف : Martine Besouw & Elena Levtchenko
- چاپ و سال / کشور: 2011
Description
Sirs, Cystinosis is a rare autosomal recessive disease caused by mutations in the CTNS gene, resulting in lysosomal accumulation of the amino acid cystine in cells throughout the body. Most patients develop general proximal tubular damage, called renal Fanconi syndrome, within the first year of life. Besides kidney involvement, the disease can manifest in the eyes, various endocrine organs, central nervous system, and muscles. If not treated with the cystine-depleting agent cysteamine, patients will develop end-stage renal disease (ESRD) around the age of 10 years. The advised dose of cysteamine is 1.30 g/m2 body surface area, with a maximum of 1.95 g/m2, divided into four equal doses. Recently, questions have been raised whether or not to decrease cysteamine dose in cystinosis patients with ESRD, as clearance of the drug might be lowered. We present a pharmacokinetic study of cysteamine in a cystinosis patient with ESRD treated with hemodialysis.
Pediatr Nephrol (2011) 26:639–640 DOI 10.1007/s00467-010-1680-1 Received: 20 September 2010 / Revised: 1 October 2010 / Accepted: 4 October 2010 / Published online: 24 October 2010
