Cysteamine hydrochloride eye drop solution for the treatment of corneal cystine crystal deposits in patients with cystinosis: an evidence-based review
نویسندگان
چکیده
Cystinosis is a rare, autosomal recessive disorder leading to defective transport of cystine out of lysosomes. Subsequent cystine crystal accumulation can occur in various tissues, including the ocular surface. This review explores the efficacy of cysteamine hydrochloride eye drops in the treatment of corneal cystine crystal accumulation and its safety profile.
منابع مشابه
A randomized clinical trial of topical cysteamine disulfide (cystamine) versus free thiol (cysteamine) in the treatment of corneal cystine crystals in cystinosis.
In nephropathic cystinosis, corneal cystine crystals cause severe photophobia and corneal erosions. Topical cysteamine dissolves these crystals, but cannot be marketed because it rapidly oxidizes to the disulfide form, cystamine, at room temperature. Since cystamine itself could be used commercially, we compared the efficacy of cystamine and cysteamine with respect to cystine crystal dissolutio...
متن کاملRemoval of corneal crystals by topical cysteamine in nephropathic cystinosis.
In patients with nephropathic cystinosis, corneal crystals develop by one year of age; they progressively accumulate and eventually cause recurrent corneal erosions and photophobia. After an in vitro study of cystinotic corneal stromal cells showed cystine depletion by cysteamine and after topical cysteamine was determined to be nontoxic in rabbits, we performed a controlled double-blind clinic...
متن کاملEvaluation of topical cysteamine therapy in the CTNS−/− knockout mouse using in vivo confocal microscopy
PURPOSE The purpose of this study was to assess the ability of quantitative in vivo confocal microscopy (CM) to detect changes in cystine crystal volume in the cystinosisn (Ctns(-/-))mouse cornea following topical cysteamine therapy. METHODS Fifteen Ctns(-/-) mice were sequentially followed using in vivo CM from 3 to 10 months of age. In a second experiment, five mice receiving topical cystea...
متن کاملA multicentre randomised double masked clinical trial of a new formulation of topical cysteamine for the treatment of corneal cystine crystals in cystinosis.
AIM To evaluate the safety and efficacy of a new topical cysteamine formulation, stable at room temperature, for the treatment of corneal cystine crystals in cystinosis. METHODS 20 study subjects were enrolled in the safety study and 16 in the efficacy study. Both studies were randomised and double blind. The primary outcome for the safety study was the occurrence of predefined serious advers...
متن کاملDrug Profile
Cystinosis is a rare autosomal recessive disorder caused by mutations in the CTNS gene located on the short arm of chromosome 17 [1,2]. The CTNS gene encodes a lysosomal cystine transporter protein, cystinosin, which helps facilitate the efflux of cystine from lysosomes. The accumulation of the disulfide amino acid cystine within cellular lysosomes leads to the formation of cystine crystals in ...
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