Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio 45229, USA.
Pediatr Neurol. 2012 Feb;46(2):83-8. doi: 10.1016/j.pediatrneurol.2011.11.020.
To determine vigabatrin's effectiveness and the prevalence of symptomatic visual impairment (i.e., impairment affecting the ability to perform everyday activities) associated with its therapy in pediatric epilepsy, we retrospectively reviewed medical records of 156 patients receiving vigabatrin at Cincinnati Children's Medical Center from 1998-2010. In addition to demographics and vigabatrin dosing information, data included seizure type/frequency at presentation and subsequent follow-up. Of 156 patients, we excluded 35 because their medical records were insufficient to permit verification of the exact duration or timing of vigabatrin treatment. To evaluate efficacy (n = 121/135), we used a 5-point scale (0-4) to compare seizure frequency at several time points. To evaluate visual impairment (n = 63), we reviewed serial ophthalmologic evaluations at baseline and during treatment for patients in whom they were clinically indicated. Mean age at treatment initiation was 1.8 years (range, 0.1-29.2 years). Treatment duration ranged from 0.7-101.0 months, with an estimated average daily dose of 79 mg/kg/day. Tuberous sclerosis complex was the commonest seizure etiology (83%). Partial-onset seizure, alone or with infantile spasms, was the commonest seizure type (84%). Seizure frequency decreased from 3.7 ± 0.6 S.D. at baseline to 1.8 ± 1.7 S.D. at 6 months (P < 0.001). Responses to vigabatrin did not differ by tuberous sclerosis complex or nontuberous sclerosis complex etiology, and were sustained for 5 years. Sixty-three patients (∼50% of all patients evaluated) underwent clinically indicated ophthalmologic assessments during the review period. In our clinical judgment, no cases of clinically relevant vigabatrin-associated visual impairment occurred. Vigabatrin was effective for refractory childhood partial-onset epilepsy, and was not associated with symptomatic vision loss.
为了确定氨己烯酸在儿科癫痫中的疗效和与其治疗相关的症状性视力障碍(即影响日常活动能力的损害)的流行率,我们回顾性分析了 1998 年至 2010 年辛辛那提儿童医院接受氨己烯酸治疗的 156 名患者的病历。除了人口统计学和氨己烯酸剂量信息外,数据还包括就诊时的癫痫发作类型/频率以及随后的随访情况。在 156 名患者中,我们排除了 35 名患者,因为他们的病历不足以证实氨己烯酸治疗的确切持续时间或时间安排。为了评估疗效(n=121/135),我们使用 5 分制(0-4)比较了几个时间点的癫痫发作频率。为了评估视力障碍(n=63),我们回顾了基线时和治疗期间临床需要的患者的连续眼科评估结果。治疗开始时的平均年龄为 1.8 岁(范围 0.1-29.2 岁)。治疗持续时间从 0.7-101.0 个月不等,平均日剂量估计为 79mg/kg/天。结节性硬化症是最常见的癫痫病因(83%)。部分性发作,单独或伴有婴儿痉挛,是最常见的癫痫类型(84%)。癫痫发作频率从基线时的 3.7±0.6 S.D.降至 6 个月时的 1.8±1.7 S.D.(P<0.001)。氨己烯酸的疗效与结节性硬化症或非结节性硬化症病因无关,并且可持续 5 年。在审查期间,63 名患者(约占所有评估患者的 50%)接受了临床需要的眼科评估。根据我们的临床判断,没有发生与氨己烯酸相关的有临床意义的视力损害病例。氨己烯酸对难治性儿童部分性癫痫有效,与症状性视力丧失无关。