Efficacy and acceptability of lanadelumab for long-term prophylaxis in hereditary angioedema: A Chinese multicenter...

Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic disorder. Lanadelumab, a monoclonal antibody inhibiting plasma kallikrein, has been approved for long-term prophylaxis (LTP) in HAE patients aged ≥12 years since 2020 in China, but real-world evidence in Chinese populations is insufficient. Here, we assessed the real-world effectiveness, safety, and treatment acceptability of lanadelumab for LTP in Chinese HAE patients.

This multicenter observational study enrolled type I/II HAE patients ≥12 years who initiated lanadelumab for LTP between June 2022 and December 2024 across 5 tertiary medical centers in China, with a minimum follow-up time of 6 months. Attack frequency, emergency interventions, patient-reported outcome measures (PROMs), dosing interval extension and acceptability were described and if feasible, compared pair-wise before and after lanadelumab treatment.

Fifty HAE patients (36 prospectively enrolled and 14 retrospectively included) were included with a median follow-up of 17.5 months. Dosing intervals were successfully extended in 80% of patients. Attack-free rate (AFR) remarkably elevated from 0.0% to 65.6% (cumulative 1-year AFR) and 66.7% (observed 1-year AFR). The proportion of patients experiencing severe attacks significantly declined. Results of PROMs also substantially improved. Treatment-emergent adverse events were mostly mild. 78% of patients reported significant economic burden associated with lanadelumab use.

Lanadelumab demonstrates high efficacy in reducing HAE attacks and improving quality of life in Chinese patients in a safe manner. Successful symptom-guided dosing interval extension was achievable in most patients. Despite clinical benefits, its high cost imposes financial burdens on our patients, but this should be weighed against the cost savings achieved through improved disease control.