Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema.

BACKGROUND:Hereditary angioedema (HAE) is associated with a substantial disease burden. Lanadelumab reduced the HAE attack rate during 132 weeks of follow-up in the HELP open-label extension (OLE) Study (NCT02741596). OBJECTIVE:To measure the impact of long-term lanadelumab treatment on patient-reported outcomes (PROs). METHODS:Rollover patients (completed the 26-week HELP study [NCT02586805]) and nonrollovers (newly enrolled) received lanadelumab 300 mg every 2 weeks. PROs (Angioedema Quality of Life Questionnaire [AE-QoL], Short Form Health Survey 12-item version 2, Hospital Anxiety and Depression Scale, Work Productivity and Activity Impairment-General Health Questionnaire, and EQ-5D-5L questionnaire) were assessed at baseline (day 0 of HELP OLE) and various time points until the end-of-study visit. The Angioedema Control Test, Treatment Satisfaction Questionnaire for Medication, and Global Impression of Treatment Response were administered starting at week 52. RESULTS:The mean (SD) change in AE-QoL total score from baseline to end-of-study for rollovers (n = 90) was -10.2 (17.9), exhibiting further improvement from HELP in health-related quality of life (HRQoL); 48.9% of rollovers achieved the previously defined 6-point minimal clinically important difference. Nonrollovers (n = 81) reported a change of -19.5 (21.3). Controlled disease (Angioedema Control Test total score ≥10) was reported by 90.2% of rollovers and 95.9% of nonrollovers at the end of the study. Excellent treatment response was reported by 78.7% of patients and 82.4% of investigators. Results from other PROs indicated a slight improvement in anxiety, a high level of satisfaction with treatment, and increased work productivityor activity. CONCLUSION:Clinically meaningful improvement in HRQoL was exhibited with long-term lanadelumab treatment, supporting the benefit of lanadelumab therapy associated with attack prevention. TRIAL REGISTRATION:ClinicalTrials.gov Identifiers: NCT02586805 (HELP Study) and NCT02741596 (HELP open-label extension).