METHODS: The HELP Study was a phase 3, randomized, double-blind, placebo-controlled study in patients with symptomatic HAE type I/II. Patients received lanadelumab 150mg q4wks (n=28), 300mg q4wks (n=29), 300mg q2wks (n=27) or placebo (n=41), for 26 weeks. The AE-QoL was administered monthly; total and domain (functioning, fatigue/mood, fear/shame, and nutrition) scores were calculated. The difference in scores from Day 0-182 was compared for placebo and the pooled and separate lanadelumab groups. Responder rates were determined by use of the AE-QoL’s minimal clinically important difference (MCID=6).
RESULTS: The pooled lanadelumab group demonstrated a significantly greater reduction in total and domain AE-QoL scores, relative to placebo (P≤0.01 for all). The largest decrease in HR-QoL impairment was observed in the functioning domain with a mean(SD) change of -29.28(22.88) for the pooled lanadelumab group vs -5.41(22.92) for placebo (P<0.01). A significantly higher proportion of patients in the pooled lanadelumab group achieved a MCID in total score (70% vs 37% for placebo, P=0.001). Lanadelumab-treated patients were 2.9 (300mg q4wks), 3.2 (150mg q4wks) and 7.2 (300mg q2wks) times more likely to achieve the MCID in total scores, compared with placebo, as assessed by regression analyses.
CONCLUSIONS: HAE patients treated with lanadelumab experienced a clinically meaningful, and statistically significant, improvement in HRQoL.