Goi Nobuhiro, Iwata Fumino, Sugihara Yoko, Higa Shingo, Chikama Taiichiro
Medical Affairs, Viatris Pharmaceuticals Japan G.K., Tokyo, Japan.
Iwata Eye Clinic, 2-15-21 Nishinakanobu, Shinagawa-ku, Tokyo, 142-0054, Japan.
Clin Ophthalmol. 2024 Nov 25;18:3457-3471. doi: 10.2147/OPTH.S479770. eCollection 2024.
Corneal cystine accumulation results in photophobia and affects patients' quality of life. We assessed the efficacy and safety of cysteamine 0.55% solution in Japanese cystinosis patients with corneal cystine crystals for 52 weeks.
This was a Phase III, open-label, single-arm study conducted in Japan (jRCT2021200029; registered on 07/12/2020). Patients with white blood cell cystine levels >1 nmol/half-cystine/mg protein or presence of corneal cystine crystal deposits identified by slit-lamp biomicroscopy were included. The primary endpoint was assessed as the in vivo confocal microscopy (IVCM) total score of 7 corneal layers. Visual acuity, photophobia and safety (adverse events [AEs]) endpoints were also evaluated.
Six patients (four males and two females) were included. The mean age (standard deviation [SD]) of the patients was 29.0 (10.30) years, with a mean treatment duration of 13.7 (0.52) months. Although the acquisition of the IVCM total score was limited, an overall downward trend was observed in IVCM scores for each layer of the cornea. A decrease in the average of smoothed intensity was observed in four out of six patients at Week 16. Most patients reported at least 1-step improvement in physician and patient-reported photophobia assessment. Thirty-three AEs were reported in five patients (83.3%). Three patients (50.0%) reported eye and general disorders and administration site conditions. Tingling sensation was the most frequently reported local adverse drug reaction. No serious AEs or deaths were reported.
Cysteamine eye drops were efficacious and well tolerated in the Japanese cystinosis patients with corneal cystine crystals.
角膜胱氨酸蓄积会导致畏光，并影响患者的生活质量。我们评估了0.55%半胱胺溶液对日本患有角膜胱氨酸结晶的胱氨酸病患者进行为期52周治疗的疗效和安全性。
这是一项在日本进行的III期开放标签单臂研究（jRCT2021200029；于2020年12月7日注册）。纳入白细胞胱氨酸水平>1 nmol/半胱氨酸/mg蛋白质或经裂隙灯生物显微镜检查发现有角膜胱氨酸晶体沉积的患者。主要终点评估为7层角膜的体内共聚焦显微镜（IVCM）总分。还评估了视力、畏光和安全性（不良事件[AE]）终点。
纳入6例患者（4例男性和2例女性）。患者的平均年龄（标准差[SD]）为29.0（10.30）岁，平均治疗持续时间为13.7（0.52）个月。尽管IVCM总分的获取有限，但观察到角膜各层的IVCM评分总体呈下降趋势。在第16周时，6例患者中有4例观察到平滑强度平均值下降。大多数患者报告在医生和患者报告的畏光评估中至少有1级改善。5例患者（83.3%）报告了33起不良事件。3例患者（50.0%）报告了眼部和全身疾病以及给药部位情况。刺痛感是最常报告的局部药物不良反应。未报告严重不良事件或死亡。
半胱胺滴眼液对患有角膜胱氨酸结晶的日本胱氨酸病患者有效且耐受性良好。
