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Case Reports
. 2008 Feb;23(2):311-5.
doi: 10.1007/s00467-007-0529-8. Epub 2007 Aug 1.

Intravenous delivery of cysteamine for the treatment of cystinosis: association with hepatotoxicity

Michael R Bendel-Stenzel  1 , Julia Steinke, Ranjan Dohil, Youngki Kim
Affiliations
Case Reports

Intravenous delivery of cysteamine for the treatment of cystinosis: association with hepatotoxicity

Michael R Bendel-Stenzel et al. Pediatr Nephrol. 2008 Feb.

Abstract

Nephropathic cystinosis is a lysosomal storage disorder, which, if untreated, results in renal failure by age 10 years. Oral cysteamine has been shown to preserve renal function in these patients. In this study, a 2-year-old girl with nephropathic cystinosis and severe gastrointestinal dysmotility was treated with intravenous (i.v.) administration of cysteamine hydrochloride (HCl). This is only the second report of long-term i.v. cysteamine therapy for nephropathic cystinosis. Unlike the treatment in the previous case, however, treatment in our patient was limited by liver toxicity.

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