治疗遗传性血管性水肿的口服药物的问世。

IF 4.6 2区 医学 Q2 ALLERGY Clinical and Translational Allergy Pub Date : 2024-09-27 DOI:10.1002/clt2.12391
Anna Valerieva, Teresa Caballero, Markus Magerl, Joao P. Frade, Paul K. Audhya, Timothy Craig
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引用次数: 0

摘要

背景:遗传性血管性水肿(HAE)是一种罕见的遗传性疾病:遗传性血管性水肿(HAE)是一种罕见的遗传性疾病,其特点是不可预测的粘膜下和/或皮下组织肿胀发作,使人衰弱,根据解剖位置的不同可能危及生命。HAE 的两种主要治疗策略是为所有患者提供有效的按需治疗,以及为适当的患者提供发作预防(短期预防 [STP] 和长期预防 [LTP])。所有获批的按需治疗药物和大多数长期预防药物都需要皮下注射或静脉注射。与注射有关的难题包括恐针症(害怕针头)、自我给药困难、注射部位反应(如疼痛、红斑、出血、瘀伤)和焦虑--所有这些都会导致依从性差和给药延迟。口服 HAE 治疗可减少治疗障碍,从而改善治疗效果。目的:回顾已批准或正在开发的用于按需治疗和/或预防 HAE 发作的口服疗法:为了提供一份全面的综述,我们通过有针对性的 PubMed 文献综述从公开资源中获取数据,并以公司网站上提供的信息作为补充(搜索截止日期为 2024 年 5 月 31 日):贝罗曲司他是一种口服血浆钙激酶(PKa)抑制剂,已被批准用于治疗LTP。另一种 PKa 抑制剂 Sebetralstat 是首个完成 3 期试验的按需口服 HAE 治疗药物。口服缓激肽 B2 受体拮抗剂 Deucrictibant 已完成按需治疗和 LTP 的 2 期试验。其他几种用于 LTP 的口服 PKa 抑制剂(ATN249、VE-4666 和 VE-4062)也处于早期开发阶段:结论:HAE口服疗法的开发已取得重大进展。这些治疗方法有望改善和优化 HAE 患者的临床疗效、满意度和生活质量。
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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Advent of oral medications for the treatment of hereditary angioedema

Background

Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable, debilitating episodes of submucosal and/or subcutaneous tissue swelling, which may be life-threatening depending on anatomic location. The two primary management strategies for HAE are ready access to effective on-demand treatment in all patients and the prevention of attacks (short-term prophylaxis [STP] and long-term prophylaxis [LTP]) in appropriate patients. All approved on-demand and most LTP medications require subcutaneous or intravenous administration. Injection-related challenges include trypanophobia (fear of needles), difficulty with self-administration, injection-site reactions (e.g., pain, erythema, bleeding, bruising), and anxiety—all contributing to poor compliance and administration delays. Oral HAE treatments may improve outcomes by reducing treatment barriers.

Aim

To review oral therapies, approved or in development, for on-demand treatment and/or prevention of HAE attacks.

Materials and Methods

To provide a comprehensive review, data was obtained from publicly available resources through a targeted PubMed literature review and supplemented by information provided on company websites (search cutoff of May 31, 2024).

Results

Berotralstat, an oral plasma kallikrein (PKa) inhibitor, is approved for LTP. Sebetralstat, another PKa inhibitor, is the investigational first oral on-demand HAE treatment to complete a phase 3 trial. Deucrictibant, an oral bradykinin B2 receptor antagonist, has completed phase 2 trials for on-demand therapy and LTP. Several other oral PKa inhibitors (ATN249, VE-4666, and VE-4062) are in early development for LTP.

Conclusion

Substantial advances have been made in the development of oral treatments for HAE. These treatments have the potential to improve and optimize clinical outcomes, satisfaction, and quality of life among patients with HAE.

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来源期刊
Clinical and Translational Allergy
Clinical and Translational Allergy Immunology and Microbiology-Immunology
CiteScore
7.50
自引率
4.50%
发文量
117
审稿时长
12 weeks
期刊介绍: Clinical and Translational Allergy, one of several journals in the portfolio of the European Academy of Allergy and Clinical Immunology, provides a platform for the dissemination of allergy research and reviews, as well as EAACI position papers, task force reports and guidelines, amongst an international scientific audience. Clinical and Translational Allergy accepts clinical and translational research in the following areas and other related topics: asthma, rhinitis, rhinosinusitis, drug hypersensitivity, allergic conjunctivitis, allergic skin diseases, atopic eczema, urticaria, angioedema, venom hypersensitivity, anaphylaxis, food allergy, immunotherapy, immune modulators and biologics, animal models of allergic disease, immune mechanisms, or any other topic related to allergic disease.
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