Cysteamine toxicity in patients with cystinosis.

Hdl Handle:
http://hdl.handle.net/10147/198704
Title:
Cysteamine toxicity in patients with cystinosis.
Authors:
Besouw, Martine T P; Bowker, Richard; Dutertre, Jean-Paul; Emma, Francesco; Gahl, William A; Greco, Marcella; Lilien, Marc R; McKiernan, John; Nobili, François; Schneider, Jerry A; Skovby, Flemming; van den Heuvel, Lambertus P; Van't Hoff, William G; Levtchenko, Elena N
Affiliation:
Department of Pediatric Nephrology, Laboratory of Pediatrics, University Hospitals Leuven, Leuven, Belgium.
Citation:
Cysteamine toxicity in patients with cystinosis. 2011, 159 (6):1004-11 J. Pediatr.
Publisher:
Elsevier
Journal:
The Journal of pediatrics
Issue Date:
Dec-2011
URI:
http://hdl.handle.net/10147/198704
DOI:
10.1016/j.jpeds.2011.05.057
PubMed ID:
21784456
Abstract:
To report new adverse effects of cysteamine.; Detailed clinical information was obtained from the patients' physicians.; 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.; 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.
Item Type:
Article
Language:
en
ISSN:
1097-6833

Full metadata record

DC FieldValue Language
dc.contributor.authorBesouw, Martine T Pen
dc.contributor.authorBowker, Richarden
dc.contributor.authorDutertre, Jean-Paulen
dc.contributor.authorEmma, Francescoen
dc.contributor.authorGahl, William Aen
dc.contributor.authorGreco, Marcellaen
dc.contributor.authorLilien, Marc Ren
dc.contributor.authorMcKiernan, Johnen
dc.contributor.authorNobili, Françoisen
dc.contributor.authorSchneider, Jerry Aen
dc.contributor.authorSkovby, Flemmingen
dc.contributor.authorvan den Heuvel, Lambertus Pen
dc.contributor.authorVan't Hoff, William Gen
dc.contributor.authorLevtchenko, Elena Nen
dc.date.accessioned2011-12-22T15:07:17Z-
dc.date.available2011-12-22T15:07:17Z-
dc.date.issued2011-12-
dc.identifier.citationCysteamine toxicity in patients with cystinosis. 2011, 159 (6):1004-11 J. Pediatr.en
dc.identifier.issn1097-6833-
dc.identifier.pmid21784456-
dc.identifier.doi10.1016/j.jpeds.2011.05.057-
dc.identifier.urihttp://hdl.handle.net/10147/198704-
dc.description.abstractTo report new adverse effects of cysteamine.-
dc.description.abstractDetailed clinical information was obtained from the patients' physicians.-
dc.description.abstractNew 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.-
dc.description.abstractCysteamine 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.-
dc.language.isoenen
dc.publisherElsevieren
dc.titleCysteamine toxicity in patients with cystinosis.en
dc.typeArticleen
dc.contributor.departmentDepartment of Pediatric Nephrology, Laboratory of Pediatrics, University Hospitals Leuven, Leuven, Belgium.en
dc.identifier.journalThe Journal of pediatricsen
dc.description.provinceMunster-

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