Shu Xiao-Mei, Li Juan, Zhang Gui-Ping, Mao Qing
Department of Pediatrics, The Affiliated Hospital, Zunyi Medical College, Zunyi, Guizhou 563003, China.
Zhongguo Dang Dai Er Ke Za Zhi. 2009 Jun;11(6):445-8.
The efficacy and adverse effects of conventional dose and low dose adrenocorticotrophic hormone (ACTH) therapy for West syndrome (WS) were compared in order to identify a low effective dose with few adverse effects.
A prospective randomized controlled study was conducted. Thirty children with cryptogenic (n=8) or symptomatic (n=22) WS were enrolled. They were randomly assigned to receive either conventional dose or low dose ACTH therapy. For the conventional dose group, ACTH 50 IU per day was administered for 2 weeks and tapered to zero over the subsequent 2 weeks. For the low dose group, 0.4 IU/kg per day was injected for 2 weeks. After seizures were fully controlled, ACTH was tapered to zero over the subsequent 2 weeks. If there was an absence of an effective response in the low dose group, the dosage was increased to 1 IU/kg per day for the next 2 weeks and then tapered to zero over 2 weeks. Both effectiveness and adverse effects were compared between the two groups.
There were no significant differences in the good initial responses between the conventional and the low dose groups, which were 53% and 60%, respectively (P> 0.05). EEG findings after ACTH therapy, the rate of relapse of spasms, and the interval to relapse were not different between the two groups (P> 0.05). The long-term outcomes were assessed in the initial 8 responders, and there were no significant differences between the two groups (follow-up duration>12 months). The rates of good efficacy and disappearance of the hypsarrhythmia were significantly higher in the cryptogenic WS group than in the symptomatic WS group (P<0.05). The incidence of ACTH therapy related-adverse effects in the conventional dose group (93%) was significantly higher than in the low dose group (20%) (P<0.01). The mild brain shrinkage was observed in one patient from the conventional dose group.
The short-term and long-term therapeutic effects of ACTH between 50 IU/d and 0.4 IU/kg/d doses are similar. ACTH therapy is more effective for cryptogenic WS than symptomatic WS. To reduce adverse effects, ACTH therapy should start with a low dose (0.4 IU/ kg each day).
比较传统剂量和低剂量促肾上腺皮质激素（ACTH）治疗韦斯特综合征（WS）的疗效和不良反应，以确定一种疗效好且不良反应少的低剂量方案。
进行一项前瞻性随机对照研究。纳入30例隐源性（n = 8）或症状性（n = 22）WS患儿。将他们随机分为接受传统剂量或低剂量ACTH治疗两组。传统剂量组，每天给予ACTH 50 IU，持续2周，随后2周逐渐减量至零。低剂量组，每天注射0.4 IU/kg，持续2周。癫痫发作完全控制后，ACTH在随后2周逐渐减量至零。如果低剂量组无有效反应，则在接下来2周将剂量增加至1 IU/kg，然后在2周内逐渐减量至零。比较两组的疗效和不良反应。
传统剂量组和低剂量组的初始良好反应率分别为53%和60%，两组之间无显著差异（P>0.05）。ACTH治疗后的脑电图结果、痉挛复发率和复发间隔在两组之间无差异（P>0.05）。对最初8例有反应者进行长期随访评估，两组之间无显著差异（随访时间>12个月）。隐源性WS组的良好疗效和高峰失律消失率显著高于症状性WS组（P<0.05）。传统剂量组ACTH治疗相关不良反应的发生率（93%）显著高于低剂量组（20%）（P<0.01）。传统剂量组有1例患者出现轻度脑萎缩。
50 IU/d和0.4 IU/kg/d剂量的ACTH的短期和长期治疗效果相似。ACTH治疗隐源性WS比症状性WS更有效。为减少不良反应，ACTH治疗应从低剂量（每天0.4 IU/kg）开始。
