Hereditary angioedema (HAE) is a rare autosomal dominant disease resulting in recurring episodes of swelling, leading to considerable patient morbidity and mortality. Lanadelumab is a plasma kallikrein inhibitor that is approved as 1st line therapy in Canada for long term prophylaxis of HAE attacks. To describe our clinical findings from a case series of adult patients with HAE type 1/2 who have been initiated on lanadelumab. A chart review of HAE type 1/2 patients at three academic centers in Canada was undertaken with demographic and clinical data extracted. Patients were included if they had been receiving lanadelumab for at least 6 months. Patients with other causes of angioedema were excluded. 12 patients meeting enrollment criteria were identified. Compared to pre-lanadelumab, patients had mean reductions of 72% and 62% in attack rate and treated attack rate respectively. 3 patients reported complete remission from attacks after starting lanadelumab. Most patients had significant improvements in HAE impact on social outings. Our case series findings support the 2019 International/Canadian HAE guideline that lanadelumab is an effective therapy for long term prophylaxis. In our patient population, initiation of lanadelumab improved disease control, minimized the burden of treatment and improved HAE impact on social outings.

中文翻译：

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lanadelumab 对遗传性血管性水肿的影响：加拿大 12 名患者的病例系列


遗传性血管性水肿 (HAE) 是一种罕见的常染色体显性遗传疾病，会导致反复发作的肿胀，从而导致相当高的患者发病率和死亡率。 Lanadelumab 是一种血浆激肽释放酶抑制剂，在加拿大被批准作为长期预防 HAE 发作的一线疗法。描述我们对已开始接受 Lanadelumab 治疗的 1/2 型 HAE 成年患者的病例系列的临床发现。对加拿大三个学术中心的 HAE 1/2 型患者进行了图表审查，并提取了人口统计和临床数据。如果患者已接受 lanadelumab 治疗至少 6 个月，则纳入研究。排除其他原因引起血管性水肿的患者。确定了 12 名符合入组标准的患者。与 lanadelumab 治疗前相比，患者的发作率和治疗后的发作率平均分别降低了 72% 和 62%。 3 名患者报告在开始 Lanadelumab 后发作完全缓解。大多数患者的 HAE 对社交外出的影响显着改善。我们的病例系列研究结果支持 2019 年国际/加拿大 HAE 指南，即 lanadelumab 是一种有效的长期预防疗法。在我们的患者群体中，启动lanadelumab改善了疾病控制，最大限度地减少了治疗负担并改善了HAE对社交外出的影响。