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.
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.
Authors: Craig B Langman; Larry A Greenbaum; Minnie Sarwal; Paul Grimm; Patrick Niaudet; Georges Deschênes; Elisabeth Cornelissen; Denis Morin; Pierre Cochat; Debora Matossian; Segolene Gaillard; Mary Jo Bagger; Patrice Rioux Journal: Clin J Am Soc Nephrol Date: 2012-05-03 Impact factor: 8.237
Authors: L Gallego-Villar; Luciana Hannibal; J Häberle; B Thöny; T Ben-Omran; G K Nasrallah; Al-N Dewik; W D Kruger; H J Blom Journal: J Inherit Metab Dis Date: 2017-06-22 Impact factor: 4.982
Authors: Justine Bacchetta; Marcella Greco; Aurélia Bertholet-Thomas; François Nobili; Jozef Zustin; Pierre Cochat; Francesco Emma; Georges Boivin Journal: Bonekey Rep Date: 2016-08-17
Authors: M Besouw; L van den Heuvel; R van Eijsden; I Bongaers; L Kluijtmans; M Dewerchin; E Levtchenko Journal: J Inherit Metab Dis Date: 2013-01-31 Impact factor: 4.982
Authors: Francesco Emma; Galina Nesterova; Craig Langman; Antoine Labbé; Stephanie Cherqui; Paul Goodyer; Mirian C Janssen; Marcella Greco; Rezan Topaloglu; Ewa Elenberg; Ranjan Dohil; Doris Trauner; Corinne Antignac; Pierre Cochat; Frederick Kaskel; Aude Servais; Elke Wühl; Patrick Niaudet; William Van't Hoff; William Gahl; Elena Levtchenko Journal: Nephrol Dial Transplant Date: 2014-09 Impact factor: 5.992
Authors: Martine T P Besouw; Maria Van Dyck; David Cassiman; Kathleen J Claes; Elena N Levtchenko Journal: Pediatr Nephrol Date: 2015-05-09 Impact factor: 3.714