Biliary atresia

IF 76.9 1区 医学 Q1 MEDICINE, GENERAL & INTERNAL Nature Reviews Disease Primers Pub Date : 2024-07-11 DOI:10.1038/s41572-024-00533-x
Paul K. H. Tam, Rebecca G. Wells, Clara S. M. Tang, Vincent C. H. Lui, Maria Hukkinen, Carlos D. Luque, Paolo De Coppi, Cara L. Mack, Mikko Pakarinen, Mark Davenport
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Abstract

Biliary atresia (BA) is a progressive inflammatory fibrosclerosing disease of the biliary system and a major cause of neonatal cholestasis. It affects 1:5,000–20,000 live births, with the highest incidence in Asia. The pathogenesis is still unknown, but emerging research suggests a role for ciliary dysfunction, redox stress and hypoxia. The study of the underlying mechanisms can be conceptualized along the likely prenatal timing of an initial insult and the distinction between the injury and prenatal and postnatal responses to injury. Although still speculative, these emerging concepts, new diagnostic tools and early diagnosis might enable neoadjuvant therapy (possibly aimed at oxidative stress) before a Kasai portoenterostomy (KPE). This is particularly important, as timely KPE restores bile flow in only 50–75% of patients of whom many subsequently develop cholangitis, portal hypertension and progressive fibrosis; 60–75% of patients require liver transplantation by the age of 18 years. Early diagnosis, multidisciplinary management, centralization of surgery and optimized interventions for complications after KPE lead to better survival. Postoperative corticosteroid use has shown benefits, whereas the role of other adjuvant therapies remains to be evaluated. Continued research to better understand disease mechanisms is necessary to develop innovative treatments, including adjuvant therapies targeting the immune response, regenerative medicine approaches and new clinical tests to improve patient outcomes.

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胆道闭锁
胆道闭锁(BA)是胆道系统的一种进行性炎症纤维硬化性疾病,也是新生儿胆汁淤积症的主要病因。其发病率为1:5,000-20,000,亚洲发病率最高。其发病机制尚不清楚,但新的研究表明睫状肌功能障碍、氧化还原应激和缺氧在其中发挥了作用。对其潜在机制的研究可以按照最初损伤的可能产前时间以及损伤与产前和产后对损伤的反应之间的区别进行概念化。尽管仍是推测,但这些新兴概念、新诊断工具和早期诊断可能有助于在进行葛西肠管造口术(KPE)前进行新辅助治疗(可能针对氧化应激)。这一点尤为重要,因为只有 50-75% 的患者能及时进行 KPE,恢复胆汁流动,其中许多人随后发展为胆管炎、门静脉高压和进行性纤维化;60-75% 的患者在 18 岁之前需要进行肝移植。早期诊断、多学科管理、集中手术和对 KPE 术后并发症的优化干预可提高存活率。术后使用皮质类固醇已显示出其益处,而其他辅助疗法的作用仍有待评估。为了开发创新治疗方法,包括针对免疫反应的辅助疗法、再生医学方法和新的临床测试,以改善患者的预后,有必要继续开展研究,以更好地了解疾病机制。
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来源期刊
Nature Reviews Disease Primers
Nature Reviews Disease Primers Medicine-General Medicine
CiteScore
76.70
自引率
0.20%
发文量
75
期刊介绍: Nature Reviews Disease Primers, a part of the Nature Reviews journal portfolio, features sections on epidemiology, mechanisms, diagnosis, management, and patient quality of life. The editorial team commissions top researchers — comprising basic scientists and clinical researchers — to write the Primers, which are designed for use by early career researchers, medical students and principal investigators. Each Primer concludes with an Outlook section, highlighting future research directions. Covered medical specialties include Cardiology, Dermatology, Ear, Nose and Throat, Emergency Medicine, Endocrinology, Gastroenterology, Genetic Conditions, Gynaecology and Obstetrics, Hepatology, Haematology, Infectious Diseases, Maxillofacial and Oral Medicine, Nephrology, Neurology, Nutrition, Oncology, Ophthalmology, Orthopaedics, Psychiatry, Respiratory Medicine, Rheumatology, Sleep Medicine, and Urology.
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