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

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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.

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Acknowledgments

We would like to thank Dr. William Gahl for his assistance in obtaining and using the cysteamine, Sigma Tau for donating the cysteamine hydrochloride, and Darlette Luke, R.Ph., for preparing and testing the drug.

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Authors and Affiliations

  1. Division of Pediatric Nephrology, MMC 491, University of Minnesota Children’s Hospital, 420 Delaware St SE, Minneapolis, MN, 55455, USA

    Michael R. Bendel-Stenzel, Julia Steinke & Youngki Kim

  2. Department of Pediatrics, UCSD Medical Center, San Diego, CA, USA

    Ranjan Dohil

Authors
  1. Michael R. Bendel-Stenzel
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  2. Julia Steinke
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  3. Ranjan Dohil
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  4. Youngki Kim
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Correspondence to Michael R. Bendel-Stenzel.

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Bendel-Stenzel, M.R., Steinke, J., Dohil, R. et al. Intravenous delivery of cysteamine for the treatment of cystinosis: association with hepatotoxicity. Pediatr Nephrol 23, 311–315 (2008). https://doi.org/10.1007/s00467-007-0529-8

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  • DOI: https://doi.org/10.1007/s00467-007-0529-8

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