TY - JOUR
T1 - Cysteamine toxicity in patients with cystinosis
AU - Besouw, Martine T.P.
AU - Bowker, Richard
AU - Dutertre, Jean Paul
AU - Emma, Francesco
AU - Gahl, William A.
AU - Greco, Marcella
AU - Lilien, Marc R.
AU - McKiernan, John
AU - Nobili, Franois
AU - Schneider, Jerry A.
AU - Skovby, Flemming
AU - Van Den Heuvel, Lambertus P.
AU - Van'T Hoff, William G.
AU - Levtchenko, Elena N.
N1 - Funding Information:
Supported by the Cystinosis Research Foundation . E.L. is supported by the Foundation for Scientific Research Flanders (grant 1801110N ). The sponsors had no role in the study design; the collection, analysis and interpretation of data; the writing of the report; or the decision to submit the manuscript for publication. The authors declare no conflicts of interest.
PY - 2011/12
Y1 - 2011/12
N2 - 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.
AB - 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.
UR - https://www.scopus.com/pages/publications/80755133423
U2 - 10.1016/j.jpeds.2011.05.057
DO - 10.1016/j.jpeds.2011.05.057
M3 - Article
C2 - 21784456
SN - 0022-3476
VL - 159
SP - 1004
EP - 1011
JO - Journal of pediatrics
JF - Journal of pediatrics
IS - 6
ER -