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

Michael R. Bendel-Stenzel, Julia Steinke, Ranjan Dohil, Youngki Kim

Research output: Contribution to journalArticle

1 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)311-315
Number of pages5
JournalPediatric Nephrology
Volume23
Issue number2
DOIs
StatePublished - Feb 1 2008

Keywords

  • Drug-induced hepatitis
  • Gastrointestinal dysmotility
  • Hypothyroidism
  • Nephropathic cystinosis
  • Renal failure

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