Effectiveness and Safety of Lanadelumab in the Treatment of Patients with Hereditary Angioedema in Routine Clinical Practice in the Russian Federation.

Le résumé fourni par la source

Background: Hereditary angioedema (HAE) caused by C1 inhibitor deficiency is a rare condition marked by recurrent swelling of the skin and mucous membranes. This study evaluated the effectiveness and safety of lanadelumab in routine clinical practice in Russia (IISR-2021-200085). Methods: A prospective cohort of 30 individuals with HAE was observed. Most patients were women (87%), with a mean age of 34 years; Seventeen percent of participants were adolescents. Prior to initiating lanadelumab, 16 patients (53%) had been receiving long-term prophylaxis, which was discontinued once lanadelumab was started. Treatment outcomes were assessed by comparing attack frequency, medication use for acute events, disease control scores, and quality of life before and after lanadelumab therapy. Results: Before treatment, patients reported an average of 8.6 attacks per month. After 6 months of lanadelumab treatment, the mean attack frequency was reduced to 0.5 per month (p < 0.001). Use of acute therapies decreased from 4.73 to 0.43 doses per month (p < 0.001). Disease control improved substantially, and quality-of-life scores improved from 58.2 to 25.3 points (p < 0.001). No significant lanadelumab-related adverse events were observed. Conclusion: In routine clinical practice, lanadelumab proved highly effective in reducing attack rates, enhancing disease control, and improving quality of life in patients with hereditary angioedema. The therapy was well tolerated, with no serious safety concerns identified. These findings reinforce lanadelumab as a reliable long-term prophylactic option for hereditary angioedema management.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.