Sažetak sa skupa
Efficacy of plasma kallikrein inhibitors in the long-term prophylaxis of hereditary angioedema in patients treated at the Otorhinolaryngology Department of Šibenik-Knin County General Hospital
Ljerka Čulav
; OB Šibensko-kninske županije, Stjepana Radića 83, 22000 Šibenik, Hrvatska
Jakov Bilać
; OB Šibensko-kninske županije, Stjepana Radića 83, 22000 Šibenik, Hrvatska
Marijeta Maričević Smolić
; OB Šibensko-kninske županije, Stjepana Radića 83, 22000 Šibenik, Hrvatska
Sažetak
Hereditary angioedema (HAE) is a rare genetic disorder characterized by the swelling of the extremities,
genitals, and abdomen, as well as a potentially life-threatening swelling of the upper airways. Depending on
the mutated gene and the concentration and activity of the C1 inhibitor, several types of the disease are
distinguished, all sharing a similar clinical presentation and treatment approach. To prevent angioedema (AE)
attacks, patients with high disease activity (swelling of the upper airways or abdomen three or more times per
month) require long-term prophylaxis (LTP), while patients with mild disease activity only require treatment
with specific therapies (C1 inhibitor concentrates, bradykinin inhibitors) during acute attacks. Until June 2024,
only attenuated androgens were available for LTP in Croatia, which in women caused side effects such as
permanent voice changes, virilization, and others. Since then, modern drugs for LTP, plasma kallikrein
inhibitors (PKI), have also become available. We examined the efficacy of PKI in patients with a previously
confirmed diagnosis of HAE type 1 (low concentration and function of C1 inhibitor, SERPING1 gene
mutation) treated at the ENT Department of Šibenik-Knin County General Hospital (GH). We analyzed the
disease activity in eight patients with HAE who had already been receiving LTP with attenuated androgens for
at least four months (according to the Croatian Health Insurance Fund criteria for the use of high-cost
medicines) and identified four female patients with high disease activity (all had confirmed upper airway
swelling) who met the criteria for switching to LTP with PKI. Three patients received lanadelumab, a
monoclonal antibody for subcutaneous administration once monthly during the first four months and, after the
reevaluation of efficacy, once monthly. One patient had been receiving therapy for the past 14 months, and the
other two for five months. During that time, they experienced no breakthrough AE attacks. The fourth patient
had been taking berotralstat tablets once daily for the past four months and experienced two breakthrough
attacks, which was considered to meet the criteria for the continuation of therapy. Apart from mild redness at
the injection site and mild nausea during the first 10 days of treatment in the patient on oral therapy, no serious
side effects have been observed.
Ključne riječi
angioedema; lanadelumab; berotralstat
Hrčak ID:
336289
URI
Datum izdavanja:
10.11.2025.
Posjeta: 154 *