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J Pediatr. 2011 Dec;159(6):1004-11. doi: 10.1016/j.jpeds.2011.05.057. Epub 2011 Jul 23.

Cysteamine toxicity in patients with cystinosis.

Author information

1
Department of Pediatric Nephrology, Laboratory of Pediatrics, University Hospitals Leuven, Leuven, Belgium.

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.

PMID:
21784456
DOI:
10.1016/j.jpeds.2011.05.057
[Indexed for MEDLINE]

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