title: A randomised placebo-controlled trial of topical cysteamine therapy in patients with nephropathic cystinosis.
abstract: Five patients with nephropathic cystinosis were evaluated to assess the ability of topical cysteamine to clear corneal cystine crystals. All patients were randomised to receive topical cysteamine 0.2% six times a day in one eye with normal saline in the other eye as a control. All five patients showed some improvement in visual symptoms (photophobia, blepharospasm and visual acuity) together with an improvement in corneal crystal density. Three of these also had an improvement in Snellen visual acuity and contrast sensitivity.
authors.affiliation: Department of Ophthalmology, St James University Hospital, Leeds.
authors.forename: J A
authors.initials: JA
authors.lastname: Bradbury
authors.forename: J P
authors.initials: JP
authors.lastname: Danjoux
authors.forename: J
authors.initials: J
authors.lastname: Voller
authors.forename: M
authors.initials: M
authors.lastname: Spencer
authors.forename: T
authors.initials: T
authors.lastname: Brocklebank
mesh_terms: D000293:Adolescent; D002648:Child; D015350:Contrast Sensitivity / Q000187:drug effects; D003315:Cornea / Q000378:metabolism; D003543:Cysteamine / Q000008:administration & dosage* / Q000494:pharmacology; D003553:Cystine / Q000378:metabolism; D003554:Cystinosis / Q000188:drug therapy* / Q000503:physiopathology; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D007676:Kidney Failure, Chronic / Q000150:complications*; D008297:Male; D009883:Ophthalmic Solutions; D014792:Visual Acuity / Q000187:drug effects
chemical_list: D009883:Ophthalmic Solutions; D003553:Cystine; D003543:Cysteamine