Askalan Rand, Mackay Mark, Brian Jessica, Otsubo Hiroshi, McDermott Catherine, Bryson Susan, Boyd Jennifer, Snead Carter, Roberts Wendy, Weiss Shelly
Division of Neurology, The Hospital for Sick Children, Toronto ON, Canada.
J Child Neurol. 2003 Mar;18(3):165-70. doi: 10.1177/08830738030180030801.
The objective of this study was to compare the efficacy of corticotropin (ACTH) versus vigabatrin in treating infantile spasms and to determine which medication has a more favorable long-term outcome in terms of cognitive function, evolution of epilepsy, and incidence of autism. Patients with infantile spasms were included in the study if they were 3 to 16 months old, had hypsarrhythmia, and had no previous treatment with vigabatrin or corticosteroids. Patient evaluation included electroencephalographic and psychometric measures before and after treatment. Patients were stratified based on etiology (idiopathic or symptomatic) and sex and then randomized between the ACTH and vigabatrin treatment groups. Each of the treatment groups received either ACTH or vigabatrin for 2 weeks. At the end of 2 weeks of treatment, patients were considered responders if spasms and hypsarrhythmia resolved. Nonresponders were crossed over and treated with the alternate drug. Nine patients were included in the study. Three patients received ACTH, one of whom was a responder. Six patients received vigabatrin, three of whom were responders. The five nonresponders received both therapies. All patients had some degree of developmental plateau or regression before the initiation of treatment. Four patients with idiopathic infantile spasms showed improved cognitive function following treatment. The remaining five patients remained significantly delayed. Five patients with symptomatic infantile spasms had epilepsy following treatment; three of them were in the autistic spectrum. The small number of infants in this pilot study is insufficient to determine which of the two drugs is more effective. However, the following trends were identified: vigabatrin may be more effective for patients with symptomatic infantile spasms; patients with idiopathic infantile spasms tend to have a better cognitive outcome; and patients with symptomatic infantile spasms tend to develop both epilepsy and autism.
本研究的目的是比较促肾上腺皮质激素（ACTH）与氨己烯酸治疗婴儿痉挛症的疗效，并确定哪种药物在认知功能、癫痫演变和自闭症发病率方面具有更有利的长期预后。年龄在3至16个月、有高峰节律紊乱且此前未接受过氨己烯酸或皮质类固醇治疗的婴儿痉挛症患者被纳入本研究。患者评估包括治疗前后的脑电图和心理测量指标。患者根据病因（特发性或症状性）和性别进行分层，然后随机分为ACTH治疗组和氨己烯酸治疗组。每个治疗组接受ACTH或氨己烯酸治疗2周。治疗2周结束时，痉挛和高峰节律紊乱消失的患者被视为有反应者。无反应者交叉使用另一种药物进行治疗。9名患者被纳入本研究。3名患者接受ACTH治疗，其中1名有反应。6名患者接受氨己烯酸治疗，其中3名有反应。5名无反应者接受了两种治疗。所有患者在治疗开始前都有一定程度的发育停滞或倒退。4名特发性婴儿痉挛症患者治疗后认知功能有所改善。其余5名患者仍有明显发育迟缓。5名症状性婴儿痉挛症患者治疗后患有癫痫；其中3名属于自闭症谱系。这项初步研究中的婴儿数量较少，不足以确定两种药物中哪种更有效。然而，发现了以下趋势：氨己烯酸可能对症状性婴儿痉挛症患者更有效；特发性婴儿痉挛症患者往往有更好的认知预后；症状性婴儿痉挛症患者往往会发展为癫痫和自闭症。
