Background: Hereditary angioedema (HAE) due to C1 inhibitor (C1INH) deficiency is characterized by recurrent attacks of edema of the skin and mucosal tissues. Symptoms usually present during childhood (mean age at first attack, 10 years). Earlier symptom onset may predict a more severe disease course. Subcutaneous (SC) C1INH is indicated for routine prophylaxis to prevent HAE attacks in adolescents and adults. We analyzed the long-term efficacy of C1INH (SC) in subjects ≤17 years old treated in an open-label extension (OLE) of the pivotal phase III Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1 Inhibitor Replacement Therapy (COMPACT) trial.

中文翻译：

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皮下注射 C1 抑制剂对儿童遗传性血管性水肿患者的长期疗效。

背景：由于 C1 抑制剂 (C1INH) 缺乏导致的遗传性血管性水肿 (HAE) 的特征是皮肤和粘膜组织水肿的反复发作。症状通常出现在儿童时期（首次发作的平均年龄为 10 岁）。较早的症状发作可能预示着更严重的病程。皮下 (SC) C1INH 适用于常规预防，以预防青少年和成人的 HAE 发作。我们分析了 C1INH (SC) 在 17 岁以下受试者中的长期疗效，该受试者在关键 III 期临床研究的开放标签扩展 (OLE) 中进行了治疗，该研究使用低容量皮下 C1 抑制剂替代疗法预防血管性水肿的最佳管理(COMPACT) 试验。