{"title":"C1 抑制剂缺乏症导致的遗传性血管性水肿:当前的治疗方法。","authors":"Giulia Costanzo, Giada Sambugaro, Davide Firinu","doi":"10.1097/ACI.0000000000001042","DOIUrl":null,"url":null,"abstract":"<p><strong>Purpose of review: </strong>For decades, treatment options for hereditary angioedema (HAE) were limited by major adverse effects, insufficient efficacy, and difficult routes of administration. However, the growing body of knowledge regarding HAE pathophysiology has led to the development of innovative drugs for self-administered, on-demand therapy and for short- and long-term prophylaxis. This review provides a comprehensive overview of the approved drugs and the development of HAE treatments.</p><p><strong>Recent findings: </strong>The implementation of new therapies will improve the application of individualized action plans based on the key goals of minimizing the number of attacks and meeting the complex needs of patients.</p><p><strong>Summary: </strong>HAE is a rare genetic disease with a high impact on patients' quality of life due to the unpredictability and variable severity of attacks. Advances in HAE research have allowed optimization of attack management and individualization of therapeutic approaches.</p>","PeriodicalId":10956,"journal":{"name":"Current Opinion in Allergy and Clinical Immunology","volume":" ","pages":"488-495"},"PeriodicalIF":3.0000,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11537475/pdf/","citationCount":"0","resultStr":"{\"title\":\"Hereditary angioedema due to C1-inhibitor deficiency: current therapeutic approaches.\",\"authors\":\"Giulia Costanzo, Giada Sambugaro, Davide Firinu\",\"doi\":\"10.1097/ACI.0000000000001042\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Purpose of review: </strong>For decades, treatment options for hereditary angioedema (HAE) were limited by major adverse effects, insufficient efficacy, and difficult routes of administration. However, the growing body of knowledge regarding HAE pathophysiology has led to the development of innovative drugs for self-administered, on-demand therapy and for short- and long-term prophylaxis. This review provides a comprehensive overview of the approved drugs and the development of HAE treatments.</p><p><strong>Recent findings: </strong>The implementation of new therapies will improve the application of individualized action plans based on the key goals of minimizing the number of attacks and meeting the complex needs of patients.</p><p><strong>Summary: </strong>HAE is a rare genetic disease with a high impact on patients' quality of life due to the unpredictability and variable severity of attacks. Advances in HAE research have allowed optimization of attack management and individualization of therapeutic approaches.</p>\",\"PeriodicalId\":10956,\"journal\":{\"name\":\"Current Opinion in Allergy and Clinical Immunology\",\"volume\":\" \",\"pages\":\"488-495\"},\"PeriodicalIF\":3.0000,\"publicationDate\":\"2024-12-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11537475/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Current Opinion in Allergy and Clinical Immunology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1097/ACI.0000000000001042\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/10/15 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q2\",\"JCRName\":\"ALLERGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Current Opinion in Allergy and Clinical Immunology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1097/ACI.0000000000001042","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/10/15 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"ALLERGY","Score":null,"Total":0}
引用次数: 0
摘要
综述目的:几十年来,遗传性血管性水肿(HAE)的治疗方案因不良反应大、疗效不佳和给药途径困难而受到限制。然而,随着对 HAE 病理生理学认识的不断深入,用于自控、按需治疗以及短期和长期预防的创新药物应运而生。本综述全面概述了已获批准的药物和 HAE 治疗方法的发展:摘要:HAE 是一种罕见的遗传性疾病,由于其发作的不可预测性和严重程度的多变性,对患者的生活质量影响很大。HAE 研究的进展使得发作管理和个性化治疗方法得以优化。
Hereditary angioedema due to C1-inhibitor deficiency: current therapeutic approaches.
Purpose of review: For decades, treatment options for hereditary angioedema (HAE) were limited by major adverse effects, insufficient efficacy, and difficult routes of administration. However, the growing body of knowledge regarding HAE pathophysiology has led to the development of innovative drugs for self-administered, on-demand therapy and for short- and long-term prophylaxis. This review provides a comprehensive overview of the approved drugs and the development of HAE treatments.
Recent findings: The implementation of new therapies will improve the application of individualized action plans based on the key goals of minimizing the number of attacks and meeting the complex needs of patients.
Summary: HAE is a rare genetic disease with a high impact on patients' quality of life due to the unpredictability and variable severity of attacks. Advances in HAE research have allowed optimization of attack management and individualization of therapeutic approaches.
期刊介绍:
This reader-friendly, bimonthly resource provides a powerful, broad-based perspective on the most important advances from throughout the world literature. Featuring renowned guest editors and focusing exclusively on one to three topics, every issue of Current Opinion in Allergy and Clinical Immunology delivers unvarnished, expert assessments of developments from the previous year. Insightful editorials and on-the-mark invited reviews cover key subjects such as upper airway disease; mechanisms of allergy and adult asthma; paediatric asthma and development of atopy; food and drug allergies; and immunotherapy.