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Lanadelumab to treat hereditary angioedema

机译:Lanadelumab治疗遗传性血统

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Lanadelumab is a human monoclonal antibody against plasma kallikrein indicated for prevention of attacks of hereditary angioedema (HAE). HAE is caused by SERPING1 gene mutations resulting in decreased or dysfunctional plasma protease C1 inhibitor (C1-INH) leading to a loss of inhibition of plasma kallikrein activity with subsequent cleavage of high-molecular-weight kininogen and release of bradykinin. There is a clear need for a non-plasma-derived, safe, effective and convenient prophylaxis of HAE attacks to reduce patients' daily burden of disease and disability. The percentage of patients who were attack-free for the last 16 weeks of a controlled study was 77% in the group receiving 300 mg lanadelumab every 2 weeks, compared with 3% with placebo. The most common side effects were mild injection-site reactions. Lanadelumab has the potential to change the approach from on-demand treatment to prophylaxis in HAE. Future studies will have to confirm long-term safety and efficacy of prophylactic long-term inhibition of plasma kallikrein.
机译:Lanadelumab是针对血浆Kallikrein的人单克隆抗体,表明预防遗传血管血管血症(HAE)的攻击。 Hae是由六种基因突变引起的,导致血浆蛋白酶C1抑制剂(C1-INH)降低或功能障碍导致抑制血浆Kallikrein活性的损失,随后对高分子重量激素和释放Bradykinin的释放。有明确需要非等离子体衍生,安全,有效和方便的预防海内袭击,以减少患者的疾病和残疾负担。在受控研究的过去16周内攻击的患者的百分比为77%,每2周接受300毫克Lanadelumab的77%,而安慰剂则为3%。最常见的副作用是轻度注射部位反应。 Lanadelumab有可能将从需求治疗的方法改变为Hae中的预防。未来的研究必须确认预防性长期抑制血浆Kallikrein的长期安全性和疗效。

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