Efficacy of topical cysteamine in nephropathic cystinosis

Amal Al-Hemidan, Samir S. Shoughy, Igor Kozak, Khalid F. Tabbara

Research output: Contribution to journalArticle

Abstract

Purpose 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. Methods 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. Results 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. Conclusions 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.

Original languageEnglish (US)
JournalBritish Journal of Ophthalmology
DOIs
StateAccepted/In press - Jan 5 2017

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Cystinosis
Cysteamine
Photophobia
Cystine
Ophthalmic Solutions
Light
Therapeutics
Outcome Assessment (Health Care)

ASJC Scopus subject areas

  • Ophthalmology
  • Sensory Systems
  • Cellular and Molecular Neuroscience

Cite this

Efficacy of topical cysteamine in nephropathic cystinosis. / Al-Hemidan, Amal; Shoughy, Samir S.; Kozak, Igor; Tabbara, Khalid F.

In: British Journal of Ophthalmology, 05.01.2017.

Research output: Contribution to journalArticle

Al-Hemidan, Amal ; Shoughy, Samir S. ; Kozak, Igor ; Tabbara, Khalid F. / Efficacy of topical cysteamine in nephropathic cystinosis. In: British Journal of Ophthalmology. 2017.
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abstract = "Purpose 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. Methods 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. Results 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. Conclusions 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.",
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