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

• DOI: 10.36691/rja5493
• Publication Date: 2023-06-06T13:47:03Z
• Authors: Elena A. Latysheva; Irina A. Manto; Liubov V. Aleshina; Elena N. Bobrikova; Ekaterina A. Vikt...; Elena M. Gracheva; Darya V. Demina; Daria S. Fomina; Anna Yu. Shcherbina; Tatiana V. Latysheva
• Keywords: Hereditary Angioedema; C1-inhibitor; Single Center

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