Initial Experience of Long-Term Prophylaxis with Lanadelumab for Hereditary Angioedema in China: a Clinical Observation of Four patients.

Abstract

Abstract Background Hereditary angioedema (HAE) is a rare genetic disorder caused by a deficiency or dysfunction of C1 esterase inhibitor (C1-INH), which leads to recurrent episodes of bradykininmediated edema. Lanadelumab is a plasma kallikrein inhibitor that is the only first line therapy for long term prophylaxis (LTP) of HAE attacks in China since 2021. Objectives To observe the clincial efficacy and safety of lanadelumab in Chinese patients with HAE in a small population of four patients. Method Four HAE patients from three academic centers in China was included. Demographic characteristics, clinical manifestions and life quality of HAE patients were recorded before and after LTP treatment of lanadelumab .The primary outcome is the attack frequence of symptoms, and disease-related work loss days. The secondary outcome is the improved life quality of patients. Health-related quality of life was assessed using the Dermatology Life Quality Index (DLQI) and the Angioedema Quality of Life Questionnaire (AE-QoL). Results Lanadelumab led to the reductions of 98.04% and 100% in attack rate and treated attack rate respectively. Three patients of four (75%) reported complete remission from attacks after starting the administration of lanadelumab. Two patients had significant improvements on AE-QoL and DLQI (both scores reduction rate were 100%) in early period of treatment (4 weeks and 2 weeks, respectively) and all the patients had significant improvement on missed work days/year (reduction rate was 100%). The efficacy of lanadelumab was stable during vaccination and infection with COVID-19. No treatment-related serious/severe treatment emergent adverse events (TEAEs) occured during the treatment of lanadelumab. Conclusions Our study first demonstrated the clinical efficacy of lanadelumab with highly safety in Chinese HAE patients. A reasonable dosage plan can ensure the stability of therapeutic effects of lanadelumab. Moreover, lanadelumab also play a protective role to prevent attack in the case of COVID-19 infection.