LONG-TERM EFFICACY, SAFETY, AND QUALITY OF LIFE WITH LANADELUMAB TREATMENT IN ADOLESCENTS WITH HEREDITARY ANGIOEDEMA

Abstract

<h3>Introduction</h3> In the HELP Open-Label Extension Study (OLE; NCT02741596), lanadelumab reduced attack rates by 87.4% versus baseline in patients ≥12 years old with hereditary angioedema type 1/2. Efficacy and safety of lanadelumab among patients <18 years old were analyzed. <h3>Methods</h3> HELP OLE enrolled "rollovers" (previously completed the HELP study [NCT02586805]) and "nonrollovers" (newly enrolled). Rollovers received a single dose of 300mg lanadelumab, followed by 300mg every 2 weeks (Q2W) for ≤33 months after their first attack. Nonrollovers received 300mg Q2W. Attack rates were summarized using descriptive statistics. Health-related quality of life (HRQoL) was assessed using validated instruments including the Angioedema Quality of Life Questionnaire (AE-QoL). <h3>Results</h3> 21 adolescent patients were enrolled (8 rollovers, 13 nonrollovers). 20(95.2%) completed ≥30 months' on-study. A prior history of laryngeal attacks was reported in 8(38.1%) patients. Overall, mean(SD) attack rates were reduced from 1.58(1.02) attacks/month at baseline to 0.11(0.20) during treatment (94.7% reduction). 8(38.1%) patients were attack-free during treatment. On average, 99.1% of days were attack-free (mean 27.7 days/month) during treatment. Patients reported an AE-QoL mean(SD) total score of 27.5(17.5) at baseline versus 7.5(13.2) at end-of-study, indicating achievement of clinically meaningful improvement. 12(57.1%) patients reported 251 related treatment-emergent adverse events (TEAEs); the majority pertained to the injection site, including 11(52.4%) patients who reported 227 events of injection site pain which were all mild or moderate in severity. There were no discontinuations due to TEAEs and no related serious AEs. <h3>Conclusion</h3> Lanadelumab demonstrated efficacy in preventing attacks in adolescent patients, and improved HRQoL. Most related TEAEs were injection site reactions.