Ann Allergy Asthma Immunol. 2024 Aug 9:S1081-1206(24)00489-7. doi: 10.1016/j.anai.2024.08.001. Online ahead of print.
ABSTRACT
BACKGROUND: Lanadelumab was well-tolerated and effective in preventing hereditary angioedema (HAE) attacks in the phase 3, double-blind, placebo-controlled HELP Study and subsequent open-label extension study, HELP OLE (NCT02741596).
OBJECTIVE: To evaluate outcomes from HELP OLE for adolescent patients aged 12 to 17 years.
METHODS: HELP OLE comprised patients who completed the HELP Study (rollovers) and new eligible (lanadelumab-naive) patients. Rollovers received a single dose of lanadelumab 300 mg at the last HELP Study visit (day 0). Treatment was then paused until patients experienced their first investigator-confirmed HAE attack, following which lanadelumab 300 mg was administered every 2 weeks (Q2W) for up to 33 months (4 weeks/month). Lanadelumab-naive patients received lanadelumab 300 mg Q2W from day 0. Patient-reported outcomes included Angioedema Quality of Life Questionnaire (AE-QoL). Safety was monitored throughout the study.
RESULTS: The subgroup analysis included 21 patients (8 rollovers, 13 lanadelumab-naive patients); 95.2% completed ≥ 30 months on study. Mean (SD) monthly attack rates decreased from 1.58 (1.0) at baseline to 0.11 (0.2) during treatment (mean 94.7% reduction). Eight (38.1%) patients were attack-free during treatment and, on average, 99.1% of days were attack-free (mean 27.7 days/month). Patients reported a mean (SD) AE-QoL total score of 27.5 (17.5) at baseline vs 7.5 (13.2) at end of study. Twelve (57.1%) patients reported treatment-related treatment-emergent adverse events; however, there were no treatment-related serious adverse events.
CONCLUSION: Lanadelumab provided long-term efficacy in preventing HAE attacks, was associated with clinically meaningful improvements in health-related quality of life and high levels of treatment satisfaction, and was well-tolerated in adolescent patients.
PMID:39128590 | DOI:10.1016/j.anai.2024.08.001
