title: Efficacy of topical cysteamine in nephropathic cystinosis.
abstract: The aim of this study is to evaluate the efficacy of topical cysteamine 0.55% eye drops in the treatment of corneal cystine crystal deposits in patients with nephropathic cystinosis.
  
  
  
  Thirty-two patients with nephropathic cystinosis were prospectively included in the study. Patients with corneal cystinosis were treated with topical cysteamine 0.55% eye drops. They were examined before treatment, on each monthly visit and after treatment at the last follow-up. Photophobia was classified as grade 0 (none) for no photophobia, grade 1 (mild) for photophobia in bright light, grade 2 (moderate) for photophobia in room light and grade 3 (severe) for photophobia in dim light. Corneal cystine crystals were graded as grade 0=none, grade 1=1-10 crystals/mm2, grade 2=11-50 crystals/mm2, grade 3=more than 50 crystals/mm2. The main outcome measure was evaluation of photophobia and resolution of corneal cystine crystals.
  
  
  
  There were 13 male and 19 female patients. The mean age was 8 years with an age range of 8 months to 19 years. The mean follow-up period was 4.1 years with a range of 2-8 years. Improvement of photophobia was not clinically significant in symptomatic patients. Patients displayed statistically significant worsening of corneal cystine deposits during the follow-up period.
  
  
  
  This study has shown that topical 0.55% cysteamine eye drops may have limited effects in decreasing the corneal cystine deposits in patients with severe forms of nephropathic cystinosis.
  
  
  
  NCT02766855, Results.
authors.affiliation: Habib Medical Group, Ophthalmology Department, Riyadh, Saudi Arabia.
authors.forename: Amal
authors.initials: A
authors.lastname: Al-Hemidan
authors.affiliation: Department of Ophthalmology, The Eye Center and The Eye Foundation for Research in Ophthalmology, Riyadh, Saudi Arabia.
authors.forename: Samir S
authors.initials: SS
authors.lastname: Shoughy
authors.affiliation: King Khaled Eye Specialist Hospital, Riyadh, Saudi Arabia.
authors.forename: Igor
authors.initials: I
authors.lastname: Kozak
authors.affiliation: Department of Ophthalmology, The Eye Center and The Eye Foundation for Research in Ophthalmology, Riyadh, Saudi Arabia.|Department of Ophthalmology, College of Medicine, King Saud University, Riyadh, Saudi Arabia.|The Wilmer Ophthalmological Institute of The Johns Hopkins University, School of Medicine, Baltimore, Maryland, USA.
authors.forename: Khalid F
authors.initials: KF
authors.lastname: Tabbara
mesh_terms: D000287:Administration, Topical; D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003316:Corneal Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D003543:Cysteamine / Q000008:administration & dosage*; D065104:Cystine Depleting Agents / Q000008:administration & dosage*; D003554:Cystinosis / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D009883:Ophthalmic Solutions; D020795:Photophobia / Q000503:physiopathology; D011446:Prospective Studies; D016896:Treatment Outcome; D014792:Visual Acuity / Q000502:physiology; D055815:Young Adult
chemical_list: D065104:Cystine Depleting Agents; D009883:Ophthalmic Solutions; D003543:Cysteamine
keywords: Cornea; Genetics; Treatment Medical