Effective long-term prophylaxis with lanadelumab in adolescents with hereditary angioedema: EMPOWER/ENABLE.
Publication Title
Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology
Document Type
Article
Publication Date
4-1-2025
Keywords
Humans; Adolescent; Male; Female; Child; Antibodies, Monoclonal, Humanized; Angioedemas, Hereditary; Treatment Outcome; adolescents; effectiveness; hereditary angioedema; lanadelumab; prophylaxis; safety; california; santa monica; psjmc
Abstract
BACKGROUND: Symptoms of hereditary angioedema (HAE) typically first present during childhood, but the frequency/severity of attacks often increases at puberty. Real-world data on long-term HAE prophylaxis in adolescents are limited. We report pooled data from adolescent patients enrolled in two Phase 4 studies (EMPOWER, ENABLE) evaluating the effectiveness/safety of lanadelumab (monoclonal antibody directed against plasma kallikrein) for the prevention of HAE attacks.
METHODS: Adolescent patients (aged 12 to < 18 years) with HAE-C1INH enrolled in EMPOWER and ENABLE received open-label lanadelumab 300 mg once every 2 weeks. Effectiveness outcomes were based on patient-reported assessments of on-treatment HAE attacks. Safety was assessed through the recording of treatment-emergent adverse events (TEAEs) and serious adverse events. This analysis categorized patients as "new" or "established" lanadelumab patients.
RESULTS: Thirteen new and seven established patients on lanadelumab were included. The observed monthly attack rate in new patients fell from 3.8 (mean) and 2.8 (median) during the pre-enrollment period to 0.65 (mean) and 0.21 (median) during the cumulative study period after lanadelumab initiation (84.2% and 92.9% reductions, respectively). In established patients, mean (SD) HAE attack rate (as treated) during the overall study period was 0.04 (0.03) attacks/month. Most HAE attacks were of mild/moderate severity. Nine new patients reported 42 TEAEs, mostly mild/moderate in severity, with 3 TEAEs reported as serious. Seven established patients reported 12 TEAEs (all mild/moderate and non-serious). No TEAEs were related to lanadelumab.
CONCLUSION: These data support lanadelumab's effectiveness/safety in adolescents with HAE, consistent with results from Phase 3 lanadelumab studies in mixed adult/adolescent populations.
CLINICAL TRIAL IDENTIFIERS: NCT03845400 (EMPOWER) and NCT04130191 (ENABLE).
Area of Special Interest
Women & Children
Specialty/Research Institute
Allergy & Immunology
Specialty/Research Institute
Pediatrics
DOI
10.1111/pai.70072
