Long-Term Real-World Outcomes in Patients with Hereditary Angioedema Receiving Lanadelumab for 3 or More Years.

Introduction: Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable, painful swelling attacks that significantly impair patients' quality of life (QoL). Clinical trials of lanadelumab led to its approval for long-term prophylaxis in patients with HAE; however, real-world data on long-term lanadelumab use in patients with HAE are limited. This analysis describes real-world outcomes of patients with HAE who have received lanadelumab as long-term prophylaxis for ≥3 years.

Methods: From January 2023 to January 2024, investigators collected data from the Adelphi Wave II Disease Specific Programme™, a real-world, cross-sectional survey of physicians and their patients with HAE in the USA. Physicians retrospectively reported attack frequency, attack severity, and QoL before lanadelumab initiation, at 12, 24, and 36 months post initiation, and at the time of the survey.

Results: Physicians reported data on 51 patients who had received lanadelumab for ≥3 years. Before lanadelumab initiation, physicians reported attack severity as mild in 49.0% of patients and very severe in 8.2%; at 36 months post lanadelumab initiation, 62.5% of patients experienced mild attacks and none experienced very severe attacks in the preceding year. The proportion of patients experiencing ≥1 attack per month on average decreased from 54.0% before lanadelumab initiation to 9.8% at the time of the survey. The proportion of patients with good or excellent QoL increased from 68.6% before lanadelumab initiation to 88.2% at the time of the survey.

Conclusion: In this real-world HAE study, patients treated with lanadelumab for ≥3 years experienced improvements in attack frequency, disease severity, and QoL.