小儿视神经炎(ON)是一种罕见的疾病,尚未得到很好的表征。据我们所知,儿科前瞻性结局研究(PON1)是第一项旨在评估视力(VA)结局的前瞻性研究,包括VA,复发风险,和最终诊断后2年。
美国和加拿大23个儿科眼科或神经眼科诊所的非随机观察性研究。
44名最初参加PON1(年龄3-16岁)的儿童中,共有28名(64%)完成了为期2年的研究访视。
参与者由研究者自行决定治疗。
年龄正常的单眼高对比度VA(HCVA)。次要结果包括低对比度VA(LCVA),神经影像学发现,最后的诊断。
共有28名参与者完成了2年的结局,中位入学年龄为10.3岁(范围,5-15);46%是女性,68%的患者在演示时出现单侧ON。最终的2年诊断包括孤立性ON(n=11,39%),髓鞘少突胶质细胞糖蛋白相关脱髓鞘(n=8,29%),多发性硬化症(MS)(n=4,14%),视神经脊髓炎谱系疾病(NMOSD)(n=3,11%),和急性播散性脑脊髓炎(n=2,7%)。两名参与者(7%;95%置信区间[CI],1-24)随后反复发作(加上1名未完成2年随访的参与者);所有人都有MS。另外两名参与者(7%)的未受影响的眼睛出现了新的发作。显示HCVA的平均值为最小分辨率角(logMAR)的0.81对数(~20/125),在6个月时提高到0.14logMAR(~20/25-2),1年时的0.12logMAR(〜20/25-2),和0.11logMAR(20/25-1)在2年(95%CI,-0.08至0.3[20/20+1-20/40-1])。24名参与者(79%)在2岁时具有年龄正常的VA(95%CI,60-90);21名参与者(66%)具有20/20或更好的视力。没有年龄正常VA的6名参与者有2年的NMOSD诊断(n=2名参与者,3只眼睛),MS(n=2名参与者,2只眼睛),和孤立的ON(n=2名参与者,3只眼睛)。呈现LCVA的平均值为1.45logMAR(~20/500-2),在6个月时提高到0.78logMAR(~20/125+2),0.69logMAR(〜20/100+1)在1年,和2年时的0.68logMAR(~20/100+2)(95%CI,0.48-0.88[20/50+1-20/150-1])。
尽管在演讲中表现不佳,大多数儿童在6个月时VA有显著改善,并维持在2年以上.相关的神经系统自身免疫诊断很常见。在5名(18%)参与者中发生了额外的ON发作(3次复发和2次新发发作)。
Pediatric optic neuritis (ON) is a rare disease that has not been well characterized. The Pediatric ON Prospective Outcomes
Study (PON1) was the first prospective
study to our knowledge aiming to evaluate visual acuity (VA) outcomes, including VA, recurrence risk, and final diagnosis 2 years after enrollment.
Nonrandomized observational
study at 23 pediatric ophthalmology or neuro-ophthalmology clinics in the United States and Canada.
A total of 28 (64%) of 44 children initially enrolled in PON1 (age 3-<16 years) who completed their 2-year
study visit.
Participants were treated at the investigator\'s discretion.
Age-normal monocular high-contrast VA (HCVA). Secondary outcomes included low-contrast VA (LCVA), neuroimaging findings, and final diagnoses.
A total of 28 participants completed the 2-year outcome with a median enrollment age of 10.3 years (range, 5-15); 46% were female, and 68% had unilateral ON at presentation. Final 2-year diagnoses included isolated ON (n = 11, 39%), myelin oligodendrocyte glycoprotein-associated demyelination (n = 8, 29%), multiple sclerosis (MS) (n = 4,14%), neuromyelitis optica spectrum disease (NMOSD) (n = 3, 11%), and acute disseminated encephalomyelitis (n = 2, 7%). Two participants (7%; 95% confidence interval [CI], 1-24) had subsequent recurrent ON (plus 1 participant who did not complete the 2-year visit); all had MS. Two other participants (7%) had a new episode in their unaffected eye. Mean presenting HCVA was 0.81 logarithm of the minimum angle of resolution (logMAR) (∼20/125), improving to 0.14 logMAR (∼20/25-2) at 6 months, 0.12 logMAR (∼20/25-2) at 1 year, and 0.11 logMAR (20/25-1) at 2 years (95% CI, -0.08 to 0.3 [20/20+1-20/40-1]). Twenty-four participants (79%) had age-normal VA at 2 years (95% CI, 60-90); 21 participants (66%) had 20/20 vision or better. The 6 participants without age-normal VA had 2-year diagnoses of NMOSD (n = 2 participants, 3 eyes), MS (n = 2 participants, 2 eyes), and isolated ON (n = 2 participants, 3 eyes). Mean presenting LCVA was 1.45 logMAR (∼20/500-2), improving to 0.78 logMAR (∼20/125+2) at 6 months, 0.69 logMAR (∼20/100+1) at 1 year, and 0.68 logMAR (∼20/100+2) at 2 years (95% CI, 0.48-0.88 [20/50+1-20/150-1]).
Despite poor VA at presentation, most children had marked improvement in VA by 6 months that was maintained over 2 years. Associated neurologic autoimmune diagnoses were common. Additional episodes of ON occurred in 5 (18%) of the participants (3 relapses and 2 new episodes).