Pediatric Neurology Unit, Department of Pediatrics, Postgraduate Institute of Medical Education and Research, Chandigarh, 160012, India.
Indian J Pediatr. 2020 Jan;87(1):6-11. doi: 10.1007/s12098-019-03105-0. Epub 2019 Nov 13.
To evaluate the safety, tolerability, and effectiveness of oral topiramate therapy in children with West syndrome.
The present study was designed as a prospective, observational study and was performed from July 2016 through June 2018 at a tertiary care pediatrics centre in North India. The study was approved by Institute Ethics Committee.
Data on 39 children with West syndrome were analyzed. Topiramate was used as an adjunct in 38 children who failed to hormonal therapy and/or vigabatrin and as initial monotherapy in one case. The study participants had a long treatment lag to hormonal therapy (median 2 mo, IQR 1-8), a preponderance of male sex (67%) and structural etiology (87%). Nine (23%) children had a cessation of epileptic spasms at a median dose of 3.8 mg/kg/d. However, seven children with initial response had relapses. There were no significant group differences between responders and non-responders. Overall, topiramate was well tolerated. Somnolence and lethargy with decreased oral intake were commonly observed adverse effects.
The study observed poor effectiveness of topiramate therapy, which is partially due to a long treatment lag and a high proportion of structural etiology.
评估口服托吡酯治疗婴儿痉挛症的安全性、耐受性和有效性。
本研究为前瞻性观察性研究，于 2016 年 7 月至 2018 年 6 月在印度北部的一家三级儿科中心进行。该研究得到了机构伦理委员会的批准。
对 39 例婴儿痉挛症患儿进行了数据分析。托吡酯作为 38 例对激素治疗和/或氨己烯酸治疗无效的辅助治疗药物，以及 1 例初始单药治疗药物使用。研究参与者对激素治疗的治疗延迟时间较长（中位数为 2 个月，IQR 为 1-8），男性（67%）和结构性病因（87%）占优势。9 例（23%）患儿在中位数剂量为 3.8mg/kg/d 时停止癫痫性痉挛发作。然而，7 例初始反应良好的患儿出现复发。应答者和无应答者之间无显著的组间差异。总体而言，托吡酯耐受性良好。常见的不良反应有嗜睡、昏睡和食欲减退。
该研究观察到托吡酯治疗的效果不佳，部分原因是治疗延迟时间长和结构性病因比例高。
