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Long-term follow-up of cystinosis patients treated with 0.55% cysteamine hydrochloride

British Journal of Ophthalmology Aug 07, 2020

Liang H, Labbé A, Baudouin C, et al. - Among 130 patients with cystinosis, a rare recessive autosomal disorder affecting lysosomal storage of the amino acid cystine, researchers tested the effectiveness, safety, and tolerability of Cystadrops (0.55% cysteamine hydrochloride in viscous solution) in the real-world clinical practice. Participants in the study were patients who received Cystadrops between 2013 and 2017 in France. Individuals received an average dosage of 3.3 (± 0.94) instillations per eye per day. Findings suggested that Cystadrops have sustained efficacy in ophthalmic parameters, maintains visual acuity, improves cystine corneal crystal scores, and improves photophobia. Corneal ulcer and keratitis were severe adverse effects but were not found to be linked to the cohort drug. This large safety cohort verifies the efficacy, safety, and tolerability of Cystadrops in real-world clinical practice.

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