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

Abstract

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. Recurrent angioedema of various localizations is the main symptom. According to the modern concept of treatment: the goal of therapy is not only to stop emerging angioedema and prevent death, but also to 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 (HAE) aged 12 years and over. A retrospective study (IISR-2021-200085) was conducted in order to evaluate its efficiency and safety in real-life practice in Russia.
 Methods: 16 patients with HAE with C1-inhibitor deficiency were enrolled at the initiation of lanadelumab treatment. The effectiveness was evaluated by comparison of patient-reported attack rate and the following PROs for the adults only were assessed (AAS, AECT, AE-QoL, HAE-AS ) initially and during the treatment. The incidence of adverse events (AEs) was evaluated.
 Results: The patients were predominantly females 81% (13/16). The average age was 29,9 years; 19% (3/16) were adolescents.
 Prior to lanadelumab, 69% (11/16) of patients received alternative long-term prophylaxis, which was cancelled after lanadelumab initiation.
 The average number of attacks/month was 10 and treated attacks/month pre-lanadelumab was 4,7 per patient. After six months of treatment these values were 0,26 and 0,09 (10 patients were totally symptom free during 6 months after the treatment start. After 3 months of treatment the mean AECT values improved from 5,6 to 14.2 (p0,001), and all patients showed an adequate disease control. After 6 months of treatment AE-QoL decreased from 58 to 19, p0,001.
 No serious AEs related to lanadelumab were observed.
 Conclusion: Our study demonstrats that lanadelumab has composite effect - minimizing the attack rate in HAE patients and improving the quality of life level. Good safety profile of lanadelumab was shown.