1 Institute of Ophthalmology, Università Cattolica del Sacro Cuore, 00168 Rome, Italy
2 Paediatric Oncology, Università Cattolica del Sacro Cuore, 00168 Rome, Italy.
Brain. 2016 Feb;139(Pt 2):404-14. doi: 10.1093/brain/awv366. Epub 2016 Jan 14.
Paediatric optic pathway gliomas are low-grade brain tumours characterized by slow progression and invalidating visual loss. Presently there is no strategy to prevent visual loss in this kind of tumour. This study evaluated the effects of nerve growth factor administration in protecting visual function in patients with optic pathway glioma-related visual impairment. A prospective randomized double-blind phase II clinical trial was conducted in 18 optic pathway glioma patients, aged from 2 to 23 years, with stable disease and severe visual loss. Ten patients were randomly assigned to receive a single 10-day course of 0.5 mg murine nerve growth factor as eye drops, while eight patients received placebo. All patients were evaluated before and after treatment, testing visual acuity, visual field, visual-evoked potentials, optic coherence tomography, electroretinographic photopic negative response, and magnetic resonance imaging. Post-treatment evaluations were repeated at 15, 30, 90, and 180 days Brain magnetic resonance imaging was performed at baseline and at 180 days. Treatment with nerve growth factor led to statistically significant improvements in objective electrophysiological parameters (electroretinographic photopic negative response amplitude at 180 days and visual-evoked potentials at 30 days), which were not observed in placebo-treated patients. Furthermore, in patients in whom visual fields could still be measured, visual field worsening was only observed in placebo-treated cases, while three of four nerve growth factor-treated subjects showed significant visual field enlargement. This corresponded to improved visually guided behaviour, as reported by the patients and/or the caregivers. There was no evidence of side effects related to nerve growth factor treatment. Nerve growth factor eye drop administration appears a safe, easy and effective strategy for the treatment of visual loss associated with optic pathway gliomas.
儿科视神经胶质瘤是一种低级别脑肿瘤，其特点是进展缓慢，并导致视力丧失。目前，对于这种肿瘤，还没有策略可以预防视力丧失。本研究评估了神经生长因子给药在保护视神经胶质瘤相关视力障碍患者的视觉功能方面的作用。在 18 名年龄在 2 至 23 岁之间、疾病稳定且存在严重视力丧失的视神经胶质瘤患者中进行了一项前瞻性随机双盲 II 期临床试验。10 名患者被随机分配接受为期 10 天的 0.5mg 鼠神经生长因子滴眼治疗，而 8 名患者接受安慰剂治疗。所有患者在治疗前后均接受评估，测试视力、视野、视觉诱发电位、光学相干断层扫描、视网膜电图明视负波和磁共振成像。治疗后 15、30、90 和 180 天重复进行评估。基线和 180 天时进行脑磁共振成像。神经生长因子治疗导致客观电生理参数的统计学显著改善（180 天的视网膜电图明视负波幅度和 30 天的视觉诱发电位），而安慰剂治疗的患者则没有观察到这些改善。此外，在仍能测量视野的患者中，仅在安慰剂治疗的病例中观察到视野恶化，而在接受神经生长因子治疗的 4 例患者中，有 3 例出现明显的视野扩大。这与患者和/或护理人员报告的视觉引导行为改善相对应。没有证据表明与神经生长因子治疗相关的副作用。神经生长因子滴眼治疗似乎是治疗视神经胶质瘤相关视力丧失的一种安全、简便、有效的策略。
