{"title":"遗传性血管性水肿的治疗进展:关注当前和未来的选择。","authors":"Kelsey Uminski, Dawn Goodyear, Stephen Betschel","doi":"10.1007/s12325-025-03382-6","DOIUrl":null,"url":null,"abstract":"<p><p>Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable and potentially life-threatening episodes of swelling, driven primarily by excessive bradykinin production. These episodes commonly involve the skin, gastrointestinal tract, and upper airway, significantly impacting patients' quality of life. Recent advances in understanding the underlying pathophysiology of HAE have transformed clinical care, enabling the development of highly targeted treatments that disrupt critical steps within the kallikrein-kinin pathway. Current on-demand therapies rapidly relieve acute symptoms, while contemporary prophylactic strategies have substantially reduced attack frequency and improved patient autonomy and health-related quality of life. Emerging therapies-including novel oral agents, monoclonal antibodies, RNA therapies, and pioneering gene editing approaches-continue to evolve, aiming to simplify treatment and further personalize care. These innovative treatments collectively strive to address remaining unmet needs, ensuring broader accessibility, convenience, and long-term sustainability of care for individuals living with HAE. This narrative review highlights the progression of therapeutic options in HAE, summarizing current advances and exploring future strategies toward personalized and patient-centered care.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":4.0000,"publicationDate":"2025-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Therapeutic Advances in Hereditary Angioedema: A Focus on Present and Future Options.\",\"authors\":\"Kelsey Uminski, Dawn Goodyear, Stephen Betschel\",\"doi\":\"10.1007/s12325-025-03382-6\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable and potentially life-threatening episodes of swelling, driven primarily by excessive bradykinin production. These episodes commonly involve the skin, gastrointestinal tract, and upper airway, significantly impacting patients' quality of life. Recent advances in understanding the underlying pathophysiology of HAE have transformed clinical care, enabling the development of highly targeted treatments that disrupt critical steps within the kallikrein-kinin pathway. Current on-demand therapies rapidly relieve acute symptoms, while contemporary prophylactic strategies have substantially reduced attack frequency and improved patient autonomy and health-related quality of life. Emerging therapies-including novel oral agents, monoclonal antibodies, RNA therapies, and pioneering gene editing approaches-continue to evolve, aiming to simplify treatment and further personalize care. These innovative treatments collectively strive to address remaining unmet needs, ensuring broader accessibility, convenience, and long-term sustainability of care for individuals living with HAE. This narrative review highlights the progression of therapeutic options in HAE, summarizing current advances and exploring future strategies toward personalized and patient-centered care.</p>\",\"PeriodicalId\":7482,\"journal\":{\"name\":\"Advances in Therapy\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":4.0000,\"publicationDate\":\"2025-10-11\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Advances in Therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s12325-025-03382-6\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"MEDICINE, RESEARCH & EXPERIMENTAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Advances in Therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s12325-025-03382-6","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
Therapeutic Advances in Hereditary Angioedema: A Focus on Present and Future Options.
Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable and potentially life-threatening episodes of swelling, driven primarily by excessive bradykinin production. These episodes commonly involve the skin, gastrointestinal tract, and upper airway, significantly impacting patients' quality of life. Recent advances in understanding the underlying pathophysiology of HAE have transformed clinical care, enabling the development of highly targeted treatments that disrupt critical steps within the kallikrein-kinin pathway. Current on-demand therapies rapidly relieve acute symptoms, while contemporary prophylactic strategies have substantially reduced attack frequency and improved patient autonomy and health-related quality of life. Emerging therapies-including novel oral agents, monoclonal antibodies, RNA therapies, and pioneering gene editing approaches-continue to evolve, aiming to simplify treatment and further personalize care. These innovative treatments collectively strive to address remaining unmet needs, ensuring broader accessibility, convenience, and long-term sustainability of care for individuals living with HAE. This narrative review highlights the progression of therapeutic options in HAE, summarizing current advances and exploring future strategies toward personalized and patient-centered care.
期刊介绍:
Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged.
The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.