Preliminary results of a non-interventional single-center study evaluating the efficacy of long-term use of lanadelumab in routine clinical practice in the Russian Federation

BACKGROUND: Hereditary angioedema with C1 inhibitor deficiency is a rare disease caused by a deficiency and/or a decrease in the functional activity of the C1 inhibitor. The primary symptom of this condition is recurrent angioedema of various localizations. According to the modern concept of treatment, the therapy aims to stop emerging angioedema and prevent death as well as achieve complete control of the disease and a high quality of life. Lanadelumab is a modern medicine developed and used to prevent attacks in patients with hereditary angioedema aged 12 years. AIM: A retrospective study (IISR-2021-200085) was conducted to evaluate the efficiency and safety of lanadelumab in real-life practice in Russia. MATERIALS AND METHODS: In all, 16 patients with hereditary angioedema and C1 inhibitor deficiency were enrolled at the initiation of lanadelumab treatment. The patients were predominantly female (81%; 13/16). The average age of patients was 29.9 years; 19% (3/16) of the patients were adolescents. The effectiveness was evaluated by comparing the patient-reported attack rates. The following PROs for the adults only were assessed initially and during the treatment: angioedema activity score, angioedema control test (AECT), angioedema quality of life questionnaire (AE-QoL), and hereditary angioedema activity score. The incidences of adverse events were evaluated. RESULTS: Before lanadelumab, 69% (11/16) of the patients received alternative long-term prophylaxis, which was canceled after the start of lanadelumab treatment. The average number of attacks per month and treated attacks per month prelanadelumab were 10 and 4.7 per patient, respectively. After 6 months of treatment, these values were 0.26 and 0.09, respectively (10 patients were symptom free at 6 months after the initiation of the treatment). After 3 months of treatment, the mean AECT values improved from 5.6 to 14.2 (p 0.001), and all patients showed adequate disease control. After 6 months of treatment, AE-QoL decreased from 58 to 19 (p 0.001). No serious adverse events related to lanadelumab were observed. CONCLUSION: Our study demonstrated that the composite effect of lanadelumab minimizes the attack rate and improves the quality of life in patients with hereditary angioedema. A good safety profile of lanadelumab is shown.