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, Francois
AU - Schneider, Jerry A.
AU - Skovby, Flemming
AU - van den Heuvel, Lambertus P.
AU - Van't Hoff, William G.
AU - Levtchenko, Elena N.
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. (J Pediatr 2011;159:1004-11).
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. (J Pediatr 2011;159:1004-11).
KW - NEPHROPATHIC CYSTINOSIS
KW - PENICILLAMINE
KW - THERAPY
KW - CHILDREN
KW - SKIN
U2 - 10.1016/j.jpeds.2011.05.057
DO - 10.1016/j.jpeds.2011.05.057
M3 - Article
SN - 0022-3476
VL - 159
SP - 1004
EP - 1011
JO - Journal of Pediatrics
JF - Journal of Pediatrics
IS - 6
ER -