Zwingelberg Sarah Barbara, Bachmann Björn O, Cursiefen Claus
Zentrum für Augenheilkunde, Universitätsklinikum Köln, Deutschland.
Klin Monbl Augenheilkd. 2020 Dec;237(12):1455-1461. doi: 10.1055/a-1274-3675. Epub 2020 Dec 7.
Topical NGF eye drops (Cenegermin) were approved in 2015 as an orphan drug for the treatment of neurotrophic keratopathy (NK). The active substance Cenegermin is a recombinant form of human NGF (nerve growth factor).
Presentation of efficacy and safety of Cenegermin for use in patients in an university real-life setting.
Retrospective study at the Köln University Eye Hospital from 2017 to 2019 with n = 11 eyes. Average follow-up was 13.5 ± 7.1 months. Reasons for treatment were neurotrophic keratopathy stage II and III, clinically mostly in combination with corneal neovascularization.
Seven eyes with a NK II and 4 eyes with a NK III with a median observation period of 13.6 months (range 1.2 - 20.3 months) from a total of 11 patients were included. The median patient age was 42.8 ± 23.6 years (range 18 - 75 years). Before the start of therapy, the median erosion area measured 3.1 ± 1.4 × 1.9 ± 1.1 mm and the median ulcer area had a size of 2.3 ± 1.1 × 2.1 ± 0.8 mm. After the start of therapy with Cenegermin (application 6×/day), the epithelial defect closed in all 11 study eyes (100%) within 4 - 12 weeks (mean: 49 d ± 9 d). In 9 out of 10 patients (90%) pre-existing corneal neovascularization regressed significantly (p < 0.001). Before the start of therapy, a value in the Luneau test of 2.9 ± 1.9 (minimum 1/6, maximum 4/6) was found and rose to a median value of 4.2 ± 1.7 (minimum 2/6, maximum 6/6) after 18 months (p = 0.015). Therapy with Cenegermin had no long-term effect on intraocular pressure: the pressure ranged between 13.2 ± 4.1 mmHg (minimum 8 mmHg, maximum 21 mmHg). Under therapy with NGF eye drops, 67% of the patients, after an initial decrease, showed a long-term improvement in visual acuity (BSCVA) from 0.72 ± 0.31 to 0.46 ± 0.29 logMAR after 18 months (p = 0.005). Relapses in form of a corneal erosion in the long-term follow-up were observed in only one eye. One patient had to stop therapy because of local pain, no other local and systemic side effects were observed.
This real-life series on the use of Cenegermin in a university context shows a good effectiveness of the substance for epithelial closure in various underlying diseases (100% within 12 weeks). There is a long-term improvement in vision and corneal sensitivity. Larger real-life cohorts with various underlying diseases should follow.
局部用神经生长因子（NGF）滴眼液（西奈吉明）于2015年被批准作为治疗神经营养性角膜病变（NK）的孤儿药。活性物质西奈吉明是重组人NGF（神经生长因子）。
介绍西奈吉明在大学实际临床环境中用于患者的疗效和安全性。
对科隆大学眼科医院2017年至2019年的11只眼睛进行回顾性研究。平均随访时间为13.5±7.1个月。治疗原因是II期和III期神经营养性角膜病变，临床上大多伴有角膜新生血管形成。
纳入了11例患者中的7只II期NK眼和4只III期NK眼，中位观察期为13.6个月（范围1.2 - 20.3个月）。患者中位年龄为42.8±23.6岁（范围18 - 75岁）。治疗开始前，糜烂面积中位数为3.1±1.4×1.9±1.1mm，溃疡面积中位数为2.3±1.1×2.1±0.8mm。开始使用西奈吉明治疗（每天滴眼6次）后，所有11只研究眼（100%）的上皮缺损在4 - 12周内（平均：49天±9天）愈合。10例患者中有9例（90%）先前存在的角膜新生血管显著消退（p<0.001）。治疗开始前，吕诺试验值为2.9±1.9（最低1/6，最高4/6），18个月后升至中位数4.2±1.7（最低2/6，最高6/6）（p = 0.015）。西奈吉明治疗对眼压无长期影响：眼压范围在13.2±4.1mmHg（最低8mmHg，最高21mmHg）之间。在使用NGF滴眼液治疗期间，67%的患者在最初视力下降后，18个月时最佳矫正视力（BSCVA）从0.72±0.31对数最小分辨角（logMAR）长期改善至0.46±0.29 logMAR（p = 0.005）。长期随访中仅一只眼出现角膜糜烂形式的复发。一名患者因局部疼痛不得不停止治疗，未观察到其他局部和全身副作用。
该关于西奈吉明在大学环境中使用的实际临床系列研究表明，该药物对各种基础疾病的上皮愈合具有良好疗效（12周内100%）。视力和角膜敏感性有长期改善。应开展更大规模的针对各种基础疾病的实际临床队列研究。
