Real-world clinical data support effectiveness and safety of lanadelumab in patients with hereditary angioedema (HAE); however, disease activity between patients can vary substantially in the absence of long-term prophylactic treatment. To assess the effectiveness of lanadelumab in patients with HAE by baseline HAE attack frequency. Patients with HAE from the phase 4 EMPOWER (NCT03845400) and ENABLE (NCT04130191) studies with available baseline attack rate data were included in this post hoc analysis. Disease activity subgroups were defined per pre-enrollment/lanadelumab (baseline) HAE attack rate (low, <1; moderate, ≥1-<2; high, ≥2-<3; very high, ≥3 attacks/month). The analysis included 152 patients (low disease activity, n = 29; moderate, n = 29; high, n = 15; very high, n = 79). In all 4 subgroups, mean and median HAE attack rates after lanadelumab initiation were low (0.0-0.5 attacks/month). Clinically meaningful improvements (≥6-point decreases) in mean Angioedema Quality of Life total scores were observed regardless of pre-lanadelumab attack rates. From month 1 after lanadelumab initiation to the end of follow-up, mean Angioedema Control Test Scores were ≥10 (indicating patient perception of well-controlled disease) in all 4 subgroups. In these real-world datasets, on-treatment lanadelumab attack rates were low regardless of baseline disease activity. Patients from all 4 subgroups experienced improvements in health-related quality of life and disease control. Overall, these findings support long-term prophylaxis with lanadelumab across disease activity levels.
Zanichelli et al. (Fri,) studied this question.