Organization of medical care for patients with hereditary angioedema and experience in treating patients with lanadelumab in the Sverdlovsk region

G. A. Bykova, Evgeny K. Beltyukov, V. Naumova, M. Karakina
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Abstract

BACKGROUND: Hereditary angioedema is a rare autosomal dominant disease that can cause edema of skin and mucous membranes, including life-threatening localization, in pathogenesis of which plasma kallikrein is also important. The main therapy aim is to achieve complete control over symptoms and improve quality of life. In Russia, the first available specialized drug for long-term prophylaxis is lanadelumab blocking plasma kallikrein. AIM: The aim is to show principles of medical care for patients with hereditary angioedema in Sverdlovsk region and to determine lanadelumab efficacy in patients with hereditary angioedema in real clinical practice. MATERIALS AND METHODS: We conducted a single-center prospective non-interventional study. There are 22 patients with hereditary angioedema in register of primary immunodeficiencies in Sverdlovsk region. The analysis of patients' therapy was performed. Lanadelumab efficacy was studied in 5 patients received drug for at least 4 months. The validated questionnaires AAS, AECT, AE-QoL were used to assess lanadelumab efficacy. RESULTS: Educational program for practitioners and implemented diagnostic algorithm led to increase hereditary angioedema detection: the number of hereditary angioedema patients doubled over past 3.5 years (11 patients in 2019, 22 patients in 2023). School for patients education was organized. Lanadelumab has been introduced into clinical practice since December 2021. All patients included in lanadelumab efficacy study were female. According to AAS28 scale, patients had high disease activity before and there was an almost 10-fold decrease in disease activity in 4 month of lanadelumab therapy. A positive effect was observed after the first month of treatment with lanadelumab. Hereditary angioedema activity decreased to 0 after 4 months of therapy. The average score on AEST scale was 5.402.41, which indicated an uncontrolled course of hereditary angioedema. By the 4th month of therapy, control was achieved ― 13.602.61 points on AEST scale. Quality of life on AE-Qol scale improved from 81% to 38% by the 4th month of therapy (p=0.050). CONCLUSION: Current system of medical care for patients with hereditary angioedema in Sverdlovsk Region makes it possible to improve hereditary angioedema detection and provide patients with special pharmacotherapy betimes. Lanadelumab significantly reduces hereditary angioedema activity, allows achieving controlled course and high quality of life in real clinical practice.
斯维尔德洛夫斯克地区遗传性血管性水肿患者的医疗护理组织和使用lanadelumab治疗患者的经验
背景:遗传性血管性水肿是一种罕见的常染色体显性遗传病,可引起皮肤和粘膜水肿,包括危及生命的局部化,其发病机制中血浆钾化因子也很重要。主要的治疗目的是达到完全控制症状和改善生活质量。在俄罗斯,第一种可用于长期预防的专门药物是lanadelumab阻断血浆钾激肽。目的:旨在展示斯维尔德洛夫斯克地区遗传性血管性水肿患者的医疗护理原则,并在实际临床实践中确定lanadelumab在遗传性血管性水肿患者中的疗效。材料和方法:我们进行了一项单中心前瞻性非干预性研究。在斯维尔德洛夫斯克地区有22例原发性免疫缺陷的遗传性血管性水肿患者。对患者的治疗进行分析。在5例接受药物治疗至少4个月的患者中研究了Lanadelumab的疗效。采用经验证的问卷AAS、AECT、AE-QoL评估lanadelumab的疗效。结果:从业人员教育计划和实施的诊断算法导致遗传性血管性水肿检出率增加:遗传性血管性水肿患者数量在过去3.5年中翻了一番(2019年11例,2023年22例)。组织患者教育学校。Lanadelumab自2021年12月起被引入临床实践。纳入lanadelumab疗效研究的所有患者均为女性。根据AAS28量表,患者之前疾病活动性高,在lanadelumab治疗4个月后疾病活动性降低了近10倍。在使用lanadelumab治疗的第一个月后观察到积极的效果。治疗4个月后,遗传性血管性水肿活性降至0。AEST评分平均为5.402.41分,提示遗传性血管性水肿病程不受控制。治疗第4个月,AEST量表- 13.602.61分达到控制。治疗第4个月时,AE-Qol量表的生活质量从81%提高到38% (p=0.050)。结论:斯维尔德洛夫斯克地区现有的遗传性血管性水肿患者医疗服务体系可提高遗传性血管性水肿的检出率,及时为患者提供特殊的药物治疗。Lanadelumab可显著降低遗传性血管性水肿活动,在实际临床实践中实现可控病程和高质量生活。
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