Long-term prophylaxis of hereditary angioedema

Hereditarni (nasljedni) angioedem (HAE) je rijetka nasljedna bolest obilježena ponavljajućim napadajima bolnog oticanja potkožnog i submukoznog tkiva. Ponavljajući napadaji angioedema dovode do funkcionalnih oštećenja, smanjenja kvalitete života (engl. Quality of Life, QoL) i povećane smrtnosti. Na temelju koncentracije i funkcije inhibitora esteraze komponente 1 (C1) komplementa (C1-INH) HAE je podijeljen u tri tipa. Tip 1 HAE (HAE-1) obilježen je niskom koncentracijom C1-INH i obuhvaća oko 85% svih slučajeva HAE. Oko 15% bolesnika s HAE ima normalnu koncentraciju C1-INH, dok je aktivnost enzima smanjena, što je obilježje tipa 2 HAE (HAE-2). Tip 3 HAE ima normalnu koncentraciju C1-INH (HAE nC1-INH). Terapija HAE uključuju liječenje akutnih napada, kratkotrajnu profilaksu (engl. short term prophylaxis, STP) i dugotrajnu profilaksu (engl. long term prophylaxis, LTP). LTP podrazumijeva primjenu terapije za smanjenje učestalosti i ozbiljnosti napadaja te poboljšanje kvalitete života u bolesnika koji imaju učestale i ozbiljne napade. U Hrvatskoj su za LTP dostupni atenuirani androgeni koji su opterećeni štetnim učincima. No, za dugoročnu profilaksu napadaja HAE moguće je primijeniti i lanadelumab. To je potpuno ljudsko monoklonsko protutijelo i vrlo je snažan i specifičan inhibitor kalikreina u plazmi, a odobren je u više zemalja EU.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