Baker James W, Bernstein Jonathan A, Harper Joseph R, Relan Anurag, Riedl Marc A
From the Allergies and Asthma, Baker Allergy Asthma Dermatology Research Center, Portland, Oregon.
Department of Internal Medicine, University of Cincinnati, Cincinnati, Ohio.
Allergy Asthma Proc. 2018 Sep 28;39(5):359-364. doi: 10.2500/aap.2018.39.4151. Epub 2018 Jun 28.
Hereditary angioedema (HAE) may occur at or spread to multiple anatomic locations during an acute attack. Recombinant human C1 esterase inhibitor (rhC1-INH) is approved for treating acute HAE attacks.
To examine the time to the beginning of symptom relief with rhC1-INH by attack location.
Data for patients ≥12 years of age with an acute HAE attack who received rhC1-INH 50 IU/kg or placebo were pooled from two double-blind clinical trials with open-label extensions. The time to the beginning of symptom relief was defined as the first time point that the visual analog scale severity score at an attack location decreased by ≥20 mm versus baseline, with persistence. Data were reported as median time values (95% confidence interval [CI]).
For abdominal attacks, the median time to the beginning of symptom relief was 60.0 minutes (95% CI, 47.0-62.0 minutes; n = 194 attacks) with rhC1-INH versus 240.0 minutes (95% CI, 45.0-720.0 minutes; n = 15 attacks) with placebo. The median time to the beginning of symptom relief for peripheral attacks was 105.0 minutes (95% CI, 90.0-120.0 minutes; n = 169 attacks) with rhC1-INH versus 303.0 minutes (95% CI, 180.0-720.0 minutes; n = 17 attacks) with placebo. For oro-facial-pharyngeal-laryngeal attacks or urogenital attacks, the median time to the beginning of symptom relief with rhC1-INH was 64.5 minutes (95% CI, 60.0-120.0 minutes; n = 36 attacks) and 119.0 minutes (95% CI, 40.0-270.0 minutes; n = 13 attacks), respectively, versus 306.0 minutes (95% CI, 30.0-495.0 minutes; n = 6 attacks) and 320.0 minutes (n = 1 attack) with placebo.
In shortening the median time to the beginning of symptom relief of acute HAE attacks, rhC1-INH 50 IU/kg was efficacious, regardless of attack location.
遗传性血管性水肿（HAE）在急性发作时可能出现在多个解剖部位或扩散至多个解剖部位。重组人C1酯酶抑制剂（rhC1-INH）已被批准用于治疗HAE急性发作。
按发作部位研究使用rhC1-INH后症状缓解开始的时间。
≥12岁的HAE急性发作患者接受50 IU/kg rhC1-INH或安慰剂治疗的数据来自两项双盲临床试验及开放标签延长期试验。症状缓解开始的时间定义为发作部位视觉模拟量表严重程度评分较基线下降≥20 mm且持续存在的第一个时间点。数据报告为中位时间值（95%置信区间[CI]）。
对于腹部发作，使用rhC1-INH症状缓解开始的中位时间为60.0分钟（95% CI，47.0 - 62.0分钟；n = 194次发作），而使用安慰剂为240.0分钟（95% CI，45.0 - 720.0分钟；n = 15次发作）。对于外周发作，使用rhC1-INH症状缓解开始的中位时间为105.0分钟（95% CI，90.0 - 120.0分钟；n = 第169次发作），而使用安慰剂为303.0分钟（95% CI，180.0 - 720.0分钟；n = 17次发作）。对于口面部 - 咽喉部发作或泌尿生殖系统发作，使用rhC1-INH症状缓解开始的中位时间分别为64.5分钟（95% CI，60.0 - 120.0分钟；n = 36次发作）和119.0分钟（95% CI，40.0 - 270.0分钟；n = 13次发作），而使用安慰剂分别为306.0分钟（95% CI，30.0 - 495.0分钟；n = 6次发作）和320.0分钟（n = 1次发作）。
50 IU/kg rhC1-INH可有效缩短HAE急性发作症状缓解开始的中位时间，无论发作部位如何。
