Cysteamine toxicity in patients with cystinosis

Martine T P Besouw, Richard Bowker, Jean Paul Dutertre, Francesco Emma, William A. Gahl, Marcella Greco, Marc R. Lilien, John McKiernan, Franois Nobili, Jerry A. Schneider, Flemming Skovby, Lambertus P. Van Den Heuvel, William G. Van'T Hoff, Elena N. Levtchenko

Research output: Contribution to journalArticlepeer-review

Abstract

Objective: To report new adverse effects of cysteamine. Study design: Detailed clinical information was obtained from the patients' physicians. Results: New adverse events were reported in 8 of 550 patients with cystinosis treated with cysteamine in Europe during the last 5 years. Detailed clinical information was not available for 2 of these patients, 1 of whom died from cerebral ischemia. The 6 evaluable patients developed vascular elbow lesions (6/6), neurologic symptoms (1/6), bone and muscle pain (2/6), and/or skin striae (2/6). Analysis of biopsy specimens from the elbow lesions demonstrated angioendotheliomatosis with irregular collagen fibers. In 3 of the 6 patients, the daily cysteamine dose exceeded the recommended maximum of 1.95 g/m 2/day. Dose reduction led to improvement of signs and symptoms in all 6 patients, suggesting a causal relationship with cysteamine administration. Conclusion: Cysteamine administration can be complicated by the development of skin, vascular, neurologic, muscular, and bone lesions. These lesions improve after cysteamine dose reduction. Doses >1.95 g/m 2/day should be prescribed with great caution, but underdosing is not advocated.

Original languageEnglish
Pages (from-to)1004-1011
Number of pages8
JournalJournal of Pediatrics
Volume159
Issue number6
DOIs
Publication statusPublished - Dec 2011

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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