College of Pharmacy, University of Georgia, Georgia Health Sciences Medical Center, Augusta, GA, USA.
Ann Pharmacother. 2013 May;47(5):744-54. doi: 10.1345/aph.1R535. Epub 2013 Apr 19.
To review the current literature on the efficacy and safety of low- versus high-dose adrenocorticotropic hormone (ACTH) regimens, low-dose ACTH regimens, and comparison of ACTH with oral corticosteroids or vigabatrin for the treatment of West syndrome.
A literature search was performed using MEDLINE, PubMed, and Inter national Pharmaceutical Abstracts (1975-November 2012) to identify studies for inclusion. In addition, reference citations from identified publications were reviewed. The following search terms were used: infantile spasms, West syndrome, adrenocorticotropic hormone, corticotropin, symptomatic West syndrome, cryptogenic West syndrome, pediatric, children, infant, adolescent, and neonate.
Studies included in this article evaluated low-dose versus high-dose ACTH, low-dose ACTH, and ACTH compared with vigabatrin and oral corticosteroids. Data reporting the efficacy and adverse effects of ACTH, vigabatrin, and oral corticosteroids were extracted from each publication. Only English-language publications were included. We initially reviewed 20 studies, and 14 were included: 5 prospective randomized clinical trials and 9 chart reviews.
West syndrome is an age-specific epileptic disorder that occurs in infancy and early childhood. It is characterized by the triad of infantile spasms, neurodevelopmental regression or delay, and hypsarrhythmia on electroencephalogram (EEG). The efficacy and adverse events of ACTH with different dosage regimens were reviewed and analyzed. ACTH compared with vigabatrin and oral corticosteroids was also evaluated. Based on this review, low-dose ACTH is probably as effective as high-dose ACTH. Compared with other agents, ACTH is suggested to be more effective than oral corticosteroids, and compared with vigabatrin, it has improved outcomes in the cessation of spasms. However, studies evaluating the efficacy of ACTH are limited by small sample size, inconsistent dosage regimens, and the use of synthetic or natural ACTH products. Serious adverse events, including intracranial hemorrhage, brain atrophy, Cushing syndrome, infection, weight gain, and hypertension, may deter the use of ACTH. Short-term therapy is recommended to reduce the risk of adverse effects.
The current literature suggests that short-term, low-dose ACTH should be considered first-line treatment of infantile spasms.
综述目前关于低剂量与高剂量促肾上腺皮质激素（ACTH）方案、低剂量 ACTH 方案治疗婴儿痉挛症的疗效和安全性，以及 ACTH 与口服皮质类固醇或氨己烯酸的比较。
使用 MEDLINE、PubMed 和国际药学文摘（1975 年-2012 年 11 月）进行文献检索以确定纳入的研究。此外，还对已确定出版物的参考文献进行了审查。使用了以下搜索词：婴儿痉挛症、West 综合征、促肾上腺皮质激素、促皮质素、症状性 West 综合征、隐源性 West 综合征、儿科、儿童、婴儿、青少年和新生儿。
本文纳入的研究评估了低剂量与高剂量 ACTH、低剂量 ACTH 以及 ACTH 与氨己烯酸和口服皮质类固醇的比较。从每项研究中提取报告 ACTH、氨己烯酸和口服皮质类固醇疗效和不良反应的数据。仅纳入英语文献。我们最初回顾了 20 项研究，其中 14 项被纳入：5 项前瞻性随机临床试验和 9 项图表回顾。
West 综合征是一种年龄特异性癫痫性疾病，发生于婴儿期和儿童早期。其特征是婴儿痉挛、神经发育退化或延迟以及脑电图（EEG）上出现高度失律。对不同剂量方案的 ACTH 的疗效和不良反应进行了回顾和分析。还评估了 ACTH 与氨己烯酸和口服皮质类固醇的比较。基于这项综述，低剂量 ACTH 可能与高剂量 ACTH 一样有效。与其他药物相比，ACTH 比口服皮质类固醇更有效，与氨己烯酸相比，它在痉挛停止方面有更好的结果。然而，评估 ACTH 疗效的研究受到样本量小、剂量方案不一致以及使用合成或天然 ACTH 产品的限制。包括颅内出血、脑萎缩、库欣综合征、感染、体重增加和高血压在内的严重不良反应可能会阻止 ACTH 的使用。建议短期治疗以降低不良反应风险。
目前的文献表明，短期、低剂量 ACTH 应作为婴儿痉挛症的一线治疗方法。
