Department of Pediatrics, Postgraduate Institute of Medical Education and Research, Chandigarh, India.
Pediatric Neurology Division, Department of Pediatrics, Postgraduate Institute of Medical Education and Research, Chandigarh, India.
Indian J Pediatr. 2021 Jul;88(7):663-667. doi: 10.1007/s12098-020-03521-7. Epub 2020 Oct 26.
To assess the feasibility, effectiveness, and safety of pulse methylprednisolone in comparison with intramuscular adrenocorticotropic hormone (ACTH) therapy in children with West syndrome (WS).
This open-label, pilot study with a parallel-group assignment included 44 recently diagnosed children with WS. Methylprednisolone therapy was given as intravenous infusion at a dose of 30 mg/kg/d for five days followed by oral steroids 1 mg/kg gradually tapered over 5-6 wk. The efficacy outcomes included a cessation of epileptic spasms (as per caregiver reporting) and resolution of hypsarrhythmia on electroencephalogram; safety outcome was the frequency of various adverse effects.
By day 14 of therapy, 6/18 (33.3%) children in the methylprednisolone group and 10/26 (38.5%) children in the ACTH group achieved cessation of epileptic spasms [group difference - 5.2%; confidence interval (CI) -30.7 to 22.8; p = 0.73]. However, by six weeks of therapy, 4/18 (22.2%) children in the methylprednisolone group and 11/26 (42.3%) children in the ACTH group had cessation of epileptic spasms (group difference - 20.1%; CI -43.0 to 8.4; p = 0.17). Hypertension was more commonly observed in the ACTH group (10 children) than in the methylprednisolone group (2 children; p = 0.046). Pulse methylprednisolone therapy was relatively safe.
The study observed limited effectiveness of both ACTH and pulse methylprednisolone therapy, which may partially be due to preponderance of structural etiology and a long treatment lag. However, pulse methylprednisolone therapy appeared to be safe, tolerable, and feasible for management of WS.
评估脉冲甲基强的松龙与肌肉注射促肾上腺皮质激素（ACTH）治疗婴儿痉挛症（WS）的可行性、有效性和安全性。
这是一项开放标签、平行分组的试点研究，纳入了 44 例新近诊断的 WS 患儿。甲基强的松龙治疗方案为静脉输注 30mg/kg/d，连用 5 天，继之以口服类固醇 1mg/kg，逐渐在 5-6 周内减量。疗效结局包括癫痫性痉挛停止（根据照料者报告）和脑电图上的高波幅失律得到缓解；安全性结局为各种不良反应的发生频率。
治疗第 14 天，甲基强的松龙组 18 例患儿中有 6 例（33.3%）和 ACTH 组 26 例患儿中有 10 例（38.5%）癫痫性痉挛停止[组间差异-5.2%；置信区间（CI）-30.7 至 22.8；p=0.73]。然而，治疗 6 周时，甲基强的松龙组 18 例患儿中有 4 例（22.2%）和 ACTH 组 26 例患儿中有 11 例（42.3%）癫痫性痉挛停止（组间差异-20.1%；CI-43.0 至 8.4；p=0.17）。ACTH 组（10 例）比甲基强的松龙组（2 例）更常出现高血压（p=0.046）。脉冲甲基强的松龙治疗相对安全。
本研究观察到 ACTH 和脉冲甲基强的松龙治疗的效果有限，这可能部分是由于结构性病因占优势和治疗延迟较长。然而，脉冲甲基强的松龙治疗似乎安全、可耐受，并且可用于 WS 的治疗。
