Lanadelumab: A Review in Hereditary Angioedema复制标题

Lanadelumab: 遗传性血管性水肿综述

Hereditary angioedema (HAE) due to C1 esterase inhibitor (C1-INH) deficiency (type 1 HAE) or dysfunction (type 2 HAE) is a rare, autosomal dominant disease caused by mutations in the SERPING1 gene, which encodes C1-INH. Plasma kallikrein (pKal) cleaves high-molecular- weight kininogen (HMWK) to generate bradykinin, which is a potent vasodilator that increases vascular permeability and leads to tissue swelling and pain associated with HAE attacks. Endogenous C1-INH regulates pKal activity; thus, C1-INH deficiency or dysfunction leads to an uncontrolled increase in pKal activity and bradykinin levels, resulting in HAE attacks.HAE attacks are managed by on-demand treatment with plasma-derived or recombinant C1-INH concentrates, a kallikrein inhibitor (ecallantide) or bradykinin-receptor antagonist (icatibant); some patients may also benefit from long-term prophylaxis.

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