Navenibart Delays Time to First Attack in Hereditary Angioedema: Results from ALPHA-STAR

Background: Hereditary angioedema (HAE) is a genetically heterogeneous autosomal dominant disorder characterized by recurrent episodes of nonpruritic, nonpitting edema increasing after puberty. It can be fatal due to laryngeal or gastrointestinal (GI) involvement with varied and changing frequency of mortality according to studies published from the Western countries. Epidemiological and clinical data of HAE in Asian countries are sparse. We sought to examine the clinical characteristics of HAE patients in Korea. Methods: Patients diagnosed with HAE at 15 tertiary hospitals across the country until 2016 were retrospectively reviewed. Results: A total of 65 patients diagnosed with HAE by 2016 were identified. The prevalence of HAE was estimated at 1.3/1,000,000 in Korea. Of the 65 patients, 21 (32.3%) were males. A total of 90.8% patients had type I HAE, while the remaining 9.2% patients had type II HAE. The first symptom developed after 20 years in 73.8% of patients, with a mean age 28.4 ± 14.1 years. The age at diagnosis was 36.5 ± 15.8 years, with a mean time delay of 7.8 ± 10.5 years. While the face (82.3%) and extremities (upper 71.0%, lower 62.9%) were the most frequently involved, the GI tract was affected in 40.5% of Korean HAE patients. Prophylaxis was maintained in 62.5% of patients. There was no reported case of death from HAE so far. Conclusions: The clinical manifestation and severity of HAE may vary according to ethnicity. HAE is more infrequent and GI involvement is less likely in Korea compared with Western countries.

Background: This study aimed to comprehensively define the clinical profile of elderly patients with hereditary angioedema (HAE) caused by C1 esterase inhibitor (C1INH) deficiency and/or dysfunction (HAE-C1INH). Furthermore, it sought to reveal age-related differences in disease expression and management by comparing these patients with their younger counterparts. Methods: In this retrospective study, seventy-six patients were included. All patients had been diagnosed with HAE-C1INH. Results: A total of 9 (12%) patients were ≥65 years, 7 (77%) of whom were female. The median age at the time of diagnosis was higher in the elderly group, whereas the median age at the first symptom was similar. There was a significant delay in diagnosis time in the elderly group. Hypertension was the most frequent comorbidity among elderly patients. The median number of angioedema attacks in the last year was 6, and similar to 10 in patients < 65 years. Angioedema control in the last three months was lower in older patients. The rate of laryngeal edema was similar in patients < 65 years and older patients. The use of short-term prophylaxis (STP) was higher in the elderly group. The most commonly used treatment for acute attacks was pdC1-INH. Two patients in the elderly group did not benefit from danazol. No adverse events with icatibant, pdC1-INH, danazol were encountered among patients. Conclusions: Compared to patients younger than 65 years of age, annual attack rates were similar, whereas elderly patients had lower angioedema control for the last three months. The use of STP rates was higher among elderly patients.