Long-term prophylaxis of hereditary angioedema

Abstract

Hereditary angioedema (HAE) is a rare inherited disease characterized by recurrent attacks of painful swelling of the subcutaneous and submucosal tissues. Recurrent attacks of angioedema lead to functional impairment, reduced quality of life (QoL) and increased mortality. Based on the concentration and function of the esterase inhibitor component 1 (C1) of complement (C1-INH) HAE is divided into three types. Type 1 HAE (HAE-1) is characterized by a low concentration of C1-INH and includes about 85% of all HAE cases. About 15% of patients with HAE have a normal concentration of C1-INH while enzyme activity is reduced, which is a feature of type 2 HAE (HAE-2). The third form of HAE has a normal concentration of C1-INH (HAE nC1-INH). HAE therapies include treatment of acute attacks, short-term prophylaxis (STP) and long-term prophylaxis (LTP). LTP involves the application of therapy to reduce the frequency and severity of attacks and improve the quality of life in patients who have frequent and severe attacks. Attenuated androgens are available for LTP in Croatia, but they are burdened with harmful effects. However, lanadelumab can also be used for long-term prophylaxis of HAE attacks. It is a fully human monoclonal antibody and is a very potent and specific inhibitor of plasma kallikrein, and has been approved in several EU countries.

Keywords

• angioedemas
• hereditary – drug therapy
• prevention and control; complement c1 inhibitor protein – therapeutic use; antibodies
• monoclonal