Berotralstat对遗传性血管水肿和获得性C1抑制剂缺乏症患者的长期预防的回顾性分析真实世界数据。

IF 8.4 2区 医学 Q1 ALLERGY Clinical Reviews in Allergy & Immunology Pub Date : 2023-12-01 Epub Date: 2023-11-02 DOI:10.1007/s12016-023-08972-2
Felix Johnson, Anna Stenzl, Benedikt Hofauer, Helen Heppt, Eva-Vanessa Ebert, Barbara Wollenberg, Robin Lochbaum, Janina Hahn, Jens Greve, Susanne Trainotti
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引用次数: 0

摘要

遗传性血管性水肿(HAE)和获得性C1抑制剂缺乏症(AAE-C1-INH)是孤儿疾病。Berotralstat是最近获得许可的长期预防(LTP),也是HAE患者的第一种口服疗法。目前还没有批准的AAE-C1-INH患者治疗方法。这项研究首次报道了接受Berotralstat治疗的AAE-C1-INH和HAE患者的真实临床数据。所有接受Berotralstat治疗的患者均纳入本项回顾性双中心研究。从患者的发作日历、治疗前以及治疗后3、6和12个月的血管性水肿生活质量(AE QoL)和血管性水肿控制测试(AECT)问卷中收集数据,然后进行分析。包括12名患者,3名AAE-C1-INH患者,7名HAE I型患者和2名HAE-nC1-INH患者。一名患者(HAE I)退出治疗。Berotralstat与所有组中较少的攻击有关。治疗6个月后,HAE I型患者(3.3至1.5)和AAE-C1-INH患者(2.3至1.0)的发作次数平均每月减少。AAE-C1-INH患者没有出现空气消化道发作。HAE-nC1-INH患者的平均值从3.8下降到1.0(3个月)。对于HAE I患者,6个月后总AE生活质量平均降低24.1分,HAE-nC1-HAE患者降低8.0分,AAE-C1-INH患者降低13.7分。6个月后,HAE I患者(平均值:7.1)、HAE-nC1-INH患者(9.0)和AAE-C1-INH患者(4.2)的AECT评分增加。接受Berotralstat治疗的HAE、HAE-nC1-INH和AAE-C1-INH患者的血管性水肿发作减少,AE生活质量和AECT评分改善。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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A Retrospective Analysis of Long-Term Prophylaxis with Berotralstat in Patients with Hereditary Angioedema and Acquired C1-Inhibitor Deficiency-Real-World Data.

Hereditary angioedema (HAE) and acquired C1-inhibitor deficiency (AAE-C1-INH) are orphan diseases. Berotralstat is a recently licensed long-term prophylaxis (LTP) and the first oral therapy for HAE patients. No approved therapies exist for AAE-C1-INH patients. This study is the first to report real-world clinical data of patients with AAE-C1-INH and HAE who received Berotralstat. All patients treated with Berotralstat were included in this retrospective, bi-centric study. Data was collected from patients' attack calendars and the angioedema quality of life (AE-QoL) and angioedema control test (AECT) questionnaires before treatment, and at 3, 6, and 12 months after treatment and was then analyzed. Twelve patients were included, 3 patients with AAE-C1-INH, 7 patients with HAE type I, and 2 patients with HAE-nC1-INH. One patient (HAE I) quit treatment. Berotralstat was associated with fewer attacks in all groups. After 6 months of treatment, a median decrease of attacks per month was noted for HAE type I patients (3.3 to 1.5) and AAE-C1-INH patients (2.3 to 1.0). No aerodigestive attacks were noted for AAE-C1-INH patients. For HAE-nC1-INH patients, a mean decrease from 3.8 to 1.0 was noted (3 months). For HAE I patients, the total AE-QoL lowered a mean of 24.1 points after 6 months, for HAE-nC1-HAE patients 8.0 points, and for AAE-C1-INH patients 13.7 points. AECT scores increased for HAE I patients (mean: 7.1), HAE-nC1-INH patients (9.0), and AAE-C1-INH patients (4.2) after 6 months. Patients with HAE, HAE-nC1-INH, and AAE-C1-INH treated with Berotralstat showed reduced angioedema attacks and improved AE-QoL and AECT scores.

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来源期刊
CiteScore
22.30
自引率
1.10%
发文量
58
审稿时长
6-12 weeks
期刊介绍: Clinical Reviews in Allergy & Immunology is a scholarly journal that focuses on the advancement of clinical management in allergic and immunologic diseases. The journal publishes both scholarly reviews and experimental papers that address the current state of managing these diseases, placing new data into perspective. Each issue of the journal is dedicated to a specific theme of critical importance to allergists and immunologists, aiming to provide a comprehensive understanding of the subject matter for a wide readership. The journal is particularly helpful in explaining how novel data impacts clinical management, along with advancements such as standardized protocols for allergy skin testing and challenge procedures, as well as improved understanding of cell biology. Ultimately, the journal aims to contribute to the improvement of care and management for patients with immune-mediated diseases.
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