OBJECTIVE To compare the effectiveness and cost-effectiveness of adrenocorticotropic hormone (ACTH) and oral steroids as first-line treatment for infantile spasm resolution, we performed a systematic review, meta-analysis, and cost-effectiveness study. METHODS A decision analysis model was populated with effectiveness data from a systematic review and meta-analysis of existing literature and cost data from publicly available prices. Effectiveness was defined as the probability of clinical spasm resolution 14 days after treatment initiation. RESULTS We included 21 studies with a total of 968 patients. The effectiveness of ACTH was not statistically significantly different from that of oral steroids (.70, 95% confidence interval [CI] = .60-.79 vs. .63, 95% CI = .56-.70; p = .28). Considering only the three available randomized trials with a total of 185 patients, the odds ratio of spasm resolution at 14 days with ACTH compared to high-dose prednisolone (4-8 mg/kg/day) was .92 (95% CI = .34-2.52, p = .87). Adjusting for potential publication bias, estimates became even more favorable to high-dose prednisolone. Using US prices, the more cost-effective treatment was high-dose prednisolone, with an incremental cost-effectiveness ratio (ICER) of $333 per case of spasms resolved, followed by ACTH, with an ICER of $1 432 200 per case of spasms resolved. These results were robust to multiple sensitivity analyses and different assumptions. Prednisolone at 4-8 mg/kg/day was more cost-effective than ACTH under a wide range of assumptions. SIGNIFICANCE For infantile spasm resolution 2 weeks after treatment initiation, current evidence does not support the preeminence of ACTH in terms of effectiveness and, especially, cost-effectiveness.

中文翻译：

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促肾上腺皮质激素与口服类固醇治疗婴儿痉挛症的成本效益

目的 为了比较促肾上腺皮质激素 (ACTH) 和口服类固醇作为治疗婴儿痉挛症的一线治疗的有效性和成本效益，我们进行了一项系统评价、荟萃分析和成本效益研究。方法 决策分析模型中填充了来自对现有文献的系统评价和元分析的有效性数据以及来自公开价格的成本数据。有效性定义为治疗开始后 14 天临床痉挛消退的可能性。结果 我们纳入了 21 项研究，共 968 名患者。ACTH 的有效性与口服类固醇的有效性无统计学差异（.70，95% 置信区间 [CI] = .60-.79 与 .63，95% CI = .56-.70；p = .28 ）。仅考虑三项共有 185 名患者的可用随机试验，ACTH 与高剂量泼尼松龙（4-8 mg/kg/天）相比，14 天痉挛缓解的优势比为 0.92（95% CI = . 34-2.52，p = .87）。调整潜在的发表偏倚后，估计值变得更加有利于高剂量泼尼松龙。使用美国价格，更具成本效益的治疗是大剂量泼尼松龙，每例痉挛得到解决的增量成本效益比 (ICER) 为 333 美元，其次是 ACTH，每例痉挛得到解决的 ICER 为 1 432 200 美元. 这些结果对于多重敏感性分析和不同假设是稳健的。在广泛的假设条件下，4-8 毫克/公斤/天的泼尼松龙比 ACTH 更具成本效益。意义 对于治疗开始 2 周后婴儿痉挛消退，