Lanadelumab Efficacy, Safety, and Injection Interval Extension in HAE: A Real-Life Study.

BACKGROUND: Lanadelumab has been available in Germany for the prophylactic treatment of hereditary angioedema since February 2019. OBJECTIVE: To investigate real-life treatment outcome of lanadelumab and gain practical experience in adapting the therapy to individual patients. METHODS: The study included 34 patients. In 24 patients with hereditary angioedema and 4 patients with angioedema due to acquired C1-inhibitor deficiency, the previous treatment was switched to lanadelumab. In 6 patients with hereditary angioedema, lanadelumab from the open-label Hereditary Angioedema Long-term Prophylaxis study was continued in regular care. During the transition, patients were monitored using the angioedema control test and the angioedema quality of life questionnaire. At the time at which patients became symptom-free, the dosage interval was increased gradually (+3 days). RESULTS: On average, the angioedema control test values improved from 7.5 (poorly controlled disease) to 14.9 (well-controlled disease), and all patients showed adequate disease control. All treated patients, except 1 outlier, scored angioedema quality of life questionnaire values representing only a slight reduction in quality of life (mean, 14 points). At the time point of data collection, 9 patients used an average fixed injection interval of 30 days. Twenty-two patients were symptom-free from the beginning of the treatment phase and intended to extend their injection interval from 30 to 32.5 days (median). We recommended reducing the initial dosing interval from 24 to 21 days (median) to 3 patients because of intermediately occurring symptoms. CONCLUSIONS: Gradual extension of injection intervals of lanadelumab presented in this study can minimize the burden of therapy without losing efficacy.