Experience of lanadelumab usage for long-term prophylaxis of attacks in hereditary angioedema in patients of the Moscow region

Abstract

BACKGROUND: Hereditary angioedema is a rare disease with high frequency for orphan diseases. The autosomal dominant type of inheritance leads to a significant number of affected patients in families. Angioedema attacks significantly impair the quality of life. hereditary angioedema causes life-threatening conditions which depend on edema’s localization. AIM: To evaluate the efficacy and safety of lanadelumab for long-term prophylaxis in hereditary angioedema in routine practice. MATERIALS AND METHODS: 16 patients with hereditary angioedema were enrolled in the observational single-center study. The data were researched: sex, patients age, age of symptoms onset, efficacy of previous methods of therapy, type of hereditary angioedema, genetics of the disease, contraindications for other prophylaxis methods. The efficacy of therapy was evaluated according to the duration of the course, the frequency of administration, the number of attacks of edema during the period of observation, and the improvement of the quality of life according to the AAS28 scale. Safety of use was evaluated by the presence of adverse events during the investigation. RESULTS: The long-term prophylaxis with lanadelumab was effectively and safety for all of 16 patients. Single attacks during of therapy were provoked by the influence of significant triggers. Life-threatening attacks were not registered. The frequency of lanadelumab injections was reduced after 12 months of usage for 50% of patients. The improvement of the quality of life (from 6 to 38 according to AAS28 scale) was confirmed for all patients. Adverse events weren’t registered during the period of treatment. CONCLUSION: The long-term prophylaxis of edema in hereditary angioedema with lanadelumab usage was approved as efficiently and safety.