Al-Hemidan Amal, Shoughy Samir S, Kozak Igor, Tabbara Khalid F
Habib Medical Group, Ophthalmology Department, Riyadh, Saudi Arabia.
Department of Ophthalmology, The Eye Center and The Eye Foundation for Research in Ophthalmology, Riyadh, Saudi Arabia.
Br J Ophthalmol. 2017 Sep;101(9):1234-1237. doi: 10.1136/bjophthalmol-2016-309278. Epub 2017 Jan 5.
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/mm, grade 2=11-50 crystals/mm, grade 3=more than 50 crystals/mm. 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.
本研究旨在评估0.55%半胱胺滴眼液治疗肾病性胱氨酸病患者角膜胱氨酸晶体沉积的疗效。
前瞻性纳入32例肾病性胱氨酸病患者。角膜胱氨酸病患者使用0.55%半胱胺滴眼液进行治疗。在治疗前、每月复诊时以及治疗后的最后一次随访时对患者进行检查。畏光程度分为0级（无），即无畏光；1级（轻度），即强光下畏光；2级（中度），即室内光线下畏光；3级（重度），即暗光下畏光。角膜胱氨酸晶体分级为0级=无，1级=1 - 10个晶体/mm，2级=11 - 50个晶体/mm，3级=超过50个晶体/mm。主要观察指标为畏光评估和角膜胱氨酸晶体的消退情况。
男性患者13例，女性患者19例。平均年龄为8岁，年龄范围为8个月至19岁。平均随访时间为4.1年，范围为2至8年。有症状患者畏光症状的改善在临床上不显著。随访期间患者角膜胱氨酸沉积有统计学意义的恶化。
本研究表明，局部使用0.55%半胱胺滴眼液对重症肾病性胱氨酸病患者减少角膜胱氨酸沉积的效果可能有限。
NCT02766855，结果。
