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Adherence to cysteamine in nephropathic cystinosis: A unique electronic monitoring experience for a better understanding. A prospective cohort study: CrYSTobs

Pediatric Nephrology Sep 12, 2020

Gaillard S, Roche L, Lemoine S, et al. - Researchers employed electronic monitoring systems to determine adherence to cysteamine in nephropathic cystinosis (NC) patients (n = 17). Participants were selected from 3 French reference centers, and included patients with verified NC, aged > 4 years and undergoing treatment with oral cysteamine (short acting or delayed release formulation as standard of care). The median daily dose of cysteamine was 1.05 (0.55–1.63) g/m2/day. For delayed release vs short acting cysteamine, the median of average number of hours covered by treatment in a day was reported to be 22.5 vs 14.9 hours. Findings demonstrated, for the first time, a rather good adherence to cysteamine, declining in adolescents and adults. A potential interest of the delayed release formulation was also reported. These observations emphasize the necessity for a multidisciplinary strategy involving therapeutic education and individualized approaches in NC patients transitioning to adulthood.

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