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

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    • Subject Terms:
      HEPATOTOXICOLOGY; CYSTINOSIS; LYSOSOMAL storage diseases; ACUTE kidney failure; KIDNEY diseases; THERAPEUTICS
    • 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. [ABSTRACT FROM AUTHOR]