Abstract

Background

The aim of the project was to identify risk factors associated with visual progression and treatment indications in pediatric patients with neurofibromatosis type 1 associated optic pathway glioma (NF1-OPG).

Methods

A multidisciplinary expert group consisting of ophthalmologists, pediatric neuro-oncologists, neurofibromatosis specialists, and neuro-radiologists involved in therapy trials assembled a cohort of children with NF1-OPG from 6 European countries with complete clinical, imaging, and visual outcome datasets. Using methods developed during a consensus workshop, visual and imaging data were reviewed by the expert team and analyzed to identify associations between factors at diagnosis with visual and imaging outcomes.

Results

Eighty-three patients (37 males, 46 females, mean age 5.1 ± 2.6 y; 1–13.1 y) registered in the European treatment trial SIOP LGG-2004 (recruited 2004–2012) were included. They were either observed or treated (at diagnosis/after follow-up).In multivariable analysis, factors present at diagnosis associated with adverse visual outcomes included: multiple visual signs and symptoms (adjusted odds ratio [adjOR]: 8.33; 95% CI: 1.9–36.45), abnormal visual behavior (adjOR: 4.15; 95% CI: 1.20–14.34), new onset of visual symptoms (adjOR: 4.04; 95% CI: 1.26–12.95), and optic atrophy (adjOR: 3.73; 95% CI: 1.13–12.53). Squint, posterior visual pathway tumor involvement, and bilateral pathway tumor involvement showed borderline significance. Treatment appeared to reduce tumor size but improved vision in only 10/45 treated patients. Children with visual deterioration after primary observation are more likely to improve with treatment than children treated at diagnosis.

Conclusions

The analysis identified the importance of symptomatology, optic atrophy, and history of vision loss as predictive factors for poor visual outcomes in children with NF1-OPG.

中文翻译：

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NF1视神经胶质瘤：分析视觉结果和治疗适应症的危险因素

摘要

背景

该项目的目的是确定与神经纤维瘤病1型相关性视神经胶质瘤（NF1-OPG）患儿有关的与视觉发展和治疗适应症相关的危险因素。

方法

由参与治疗试验的眼科医生，儿科神经肿瘤专家，神经纤维瘤病专家和神经放射科医生组成的多学科专家小组，汇集了来自六个欧洲国家的NF1-OPG儿童，他们具有完整的临床，影像和视觉结果数据集。使用在共识研讨会上开发的方法，专家组对视觉和影像数据进行了审查并进行了分析，以识别诊断因素与视觉和影像结果之间的关联。

结果

纳入了在欧洲治疗试验SIOP LGG-2004（于2004-2012年招募）中登记的八十三名患者（男37例，女46例，平均年龄5.1±2.6岁； 1-13.1岁）。在多变量分析中，诊断时与不良视觉结果相关的因素包括：多种视觉体征和症状（调整后的优势比[adjOR]：8.33； 95％CI： 1.9–36.45），视觉行为异常（adjOR：4.15； 95％CI：1.20-14-1.34），新出现的视觉症状（adjOR：4.04； 95％CI：1.26--12.95）和视神经萎缩（adjOR：3.73； 95） ％CI：1.13-12.53）。肌，后路视觉通路肿瘤受累和双侧通路肿瘤受累具有临界意义。治疗似乎减少了肿瘤的大小，但仅10/45治疗的患者改善了视力。

结论

该分析确定了症状，视神经萎缩和视力丧失史作为NF1-OPG儿童视力不良的预测因素的重要性。