Stein Eye Institute, University of California, Los Angeles.
Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland.
JAMA Ophthalmol. 2020 Dec 1;138(12):1253-1261. doi: 10.1001/jamaophthalmol.2020.4231.
Optic neuritis (ON) in children is uncommon. There are limited prospective data for visual acuity (VA) outcomes, associated diseases, and neuroimaging findings. Prospective data from a large sample would be useful for counseling families on treatment decisions and prognosis.
To prospectively study children with a first episode of ON, describe VA after 6 months, and ascertain the network's (Pediatric Eye Disease Investigator Group and Neuro-Ophthalmology Research Disease Investigator Consortium) ability to enroll pediatric patients with ON prospectively.
DESIGN, SETTING, AND PARTICIPANTS: This nonrandomized cohort study was conducted from September 20, 2016, to July 20, 2018, at 23 sites in the United States and Canada in pediatric ophthalmology or neuro-ophthalmology clinics. A total of 44 children (aged 3-15 years) presented with a first episode of ON (visual loss, pain on eye movements, or both) within 2 weeks of symptom onset and at least 1 of the following in the affected eye: a distance high-contrast VA (HCVA) deficit of at least 0.2 logMAR below age-based norms, diminished color vision, abnormal visual field, or optic disc swelling. Exclusion criteria included preexisting ocular abnormalities or a previous episode of ON.
Primary outcomes were monocular HCVA and low-contrast VA at 6 months. Secondary outcomes were neuroimaging, associated diagnoses, and antibodies for neuromyelitis optica and myelin oligodendrocyte glycoprotein.
A total of 44 children (mean age [SD], 10.2 [3.5] years; 26 boys [59%]; 23 White individuals [52%]; 54 eyes) were enrolled in the study. Sixteen patients (36%) had bilateral ON. Magnetic resonance imaging revealed white matter lesions in 23 children (52%). Of these children, 8 had myelin oligodendrocyte glycoprotein-associated demyelination (18%), 7 had acute disseminated encephalomyelitis (16%), 5 had multiple sclerosis (11%), and 3 had neuromyelitis optica (7%). The baseline mean HCVA was 0.95 logMAR (20/200), which improved by a mean 0.76 logMAR (95% CI, 0.54-0.99; range, -0.70 to 1.80) to 0.12 logMAR (20/25) at 6 months. The baseline mean distance low-contrast VA was 1.49 logMAR (20/640) and improved by a mean 0.72 logMAR (95% CI, 0.54-0.89; range, -0.20 to 1.50) to 0.73 logMAR (20/100) at 6 months. Baseline HCVA was worse in younger participants (aged <10 years) with associated neurologic autoimmune diagnoses, white matter lesions, and in those of non-White race and non-Hispanic ethnicity. The data did not suggest a statistically significant association between baseline factors and improvement in HCVA.
The study network did not reach its targeted enrollment of 100 pediatric patients with ON over 2 years. This indicates that future treatment trials may need to use different inclusion criteria or plan a longer enrollment period to account for the rarity of the disease. Despite poor VA at presentation, most children had marked improvement by 6 months. Associated neurologic autoimmune diagnoses were common. These findings can be used to counsel families about the disease.
儿童的视神经炎(ON)并不常见。对于视力(VA)预后、相关疾病和神经影像学发现,前瞻性数据有限。从大量样本中获得的前瞻性数据对于就治疗决策和预后为家庭提供咨询将非常有用。
前瞻性研究首次发作 ON 的儿童,描述 6 个月后的 VA,并确定网络(儿科眼病研究组和神经眼科研究疾病研究组)前瞻性招募患有 ON 的儿科患者的能力。
设计、地点和参与者:这项非随机队列研究于 2016 年 9 月 20 日至 2018 年 7 月 20 日在美国和加拿大的 23 个地点的儿科眼科或神经眼科诊所进行。共有 44 名儿童(年龄 3-15 岁)在症状出现后 2 周内出现首次 ON 发作(视力丧失、眼球运动疼痛或两者兼有),并且受影响的眼睛至少有以下一项:距离高对比度 VA(HCVA)至少比年龄标准低 0.2 对数 MAR,色觉减退,视野异常或视盘肿胀。排除标准包括先前存在的眼部异常或先前发生的 ON。
主要结果是 6 个月时的单眼 HCVA 和低对比度 VA。次要结果是神经影像学、相关诊断以及抗神经髓鞘抗体和髓鞘少突胶质细胞糖蛋白抗体。
共有 44 名儿童(平均年龄[标准差],10.2[3.5]岁;26 名男孩[59%];23 名白人个体[52%];54 只眼睛)入组了这项研究。16 名患者(36%)患有双侧 ON。磁共振成像显示 23 名儿童(52%)有白质病变。这些儿童中有 8 人患有髓鞘少突胶质细胞糖蛋白相关脱髓鞘(18%),7 人患有急性播散性脑脊髓炎(16%),5 人患有多发性硬化症(11%),3 人患有视神经脊髓炎(7%)。基线平均 HCVA 为 0.95 logMAR(20/200),6 个月时平均提高 0.76 logMAR(95%CI,0.54-0.99;范围,-0.70 至 1.80)至 0.12 logMAR(20/25)。基线平均远距离低对比度 VA 为 1.49 logMAR(20/640),6 个月时平均提高 0.72 logMAR(95%CI,0.54-0.89;范围,-0.20 至 1.50)至 0.73 logMAR(20/100)。基线 HCVA 在伴有神经自身免疫诊断、白质病变以及非白人种族和非西班牙裔个体的年轻参与者(<10 岁)中较差。数据并未表明基线因素与 HCVA 改善之间存在统计学显著关联。
该研究网络在 2 年内未达到其招募 100 名 ON 儿科患者的目标。这表明未来的治疗试验可能需要使用不同的纳入标准或计划更长的入组期,以考虑到该疾病的罕见性。尽管在发病时视力较差,但大多数儿童在 6 个月时都有明显改善。相关神经自身免疫诊断很常见。这些发现可以用于向家庭提供有关该疾病的咨询。