Asthma and Respiratory Research, Penn State University, 500 University Drive, Hershey, PA 17033, USA.
J Clin Immunol. 2010 Nov;30(6):823-9. doi: 10.1007/s10875-010-9442-1. Epub 2010 Jul 16.
Hereditary angioedema (HAE) is a rare disorder characterized by C1 esterase inhibitor (C1-INH) deficiency, resulting in periodic attacks of acute edema that can be life-threatening if they occur in the laryngeal region. We assessed the efficacy of C1-INH concentrate in the emergency treatment of rarely occurring acute laryngeal HAE attacks in a prospective, open-label clinical study.
Acute laryngeal attacks were each treated with C1-INH concentrate (Berinert) at a single dose of 20 U/kg body weight. Efficacy endpoints included time to onset of symptom relief and time to complete resolution of all symptoms, each based on the patient's assessment.
All 39 laryngeal attacks in 16 patients were treated successfully. The median time to onset of symptom relief was 15 min. The median time to complete resolution of all symptoms was 8.25 h. No treatment-related serious adverse events occurred, and the treatment was well tolerated. The administration of C1-INH concentrate was not associated with any viral infections.
C1-INH concentrate is an effective and safe emergency treatment for providing reliable and rapid relief from the potentially life-threatening symptoms of laryngeal HAE attacks.
遗传性血管性水肿（HAE）是一种罕见的疾病，其特征是 C1 酯酶抑制剂（C1-INH）缺乏，导致周期性急性水肿发作，如果发生在喉部，可能会危及生命。我们在一项前瞻性、开放标签的临床研究中评估了 C1-INH 浓缩物在紧急治疗罕见的急性喉部 HAE 发作中的疗效。
每次用 20U/kg 体重的 C1-INH 浓缩物（Berinert）治疗急性喉部发作。疗效终点包括症状缓解的开始时间和所有症状完全缓解的时间，均基于患者的评估。
16 名患者的 39 次喉部发作均成功治疗。症状缓解的中位数时间为 15 分钟。所有症状完全缓解的中位数时间为 8.25 小时。无治疗相关严重不良事件发生，且治疗耐受性良好。C1-INH 浓缩物的给药与任何病毒感染无关。
C1-INH 浓缩物是一种有效且安全的紧急治疗方法，可可靠且快速缓解喉部 HAE 发作的潜在危及生命的症状。
