Erschienen in:
01.02.2008 | Brief Report
Intravenous delivery of cysteamine for the treatment of cystinosis: association with hepatotoxicity
verfasst von:
Michael R. Bendel-Stenzel, Julia Steinke, Ranjan Dohil, Youngki Kim
Erschienen in:
Pediatric Nephrology
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Ausgabe 2/2008
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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.