Old and novel prognostic biomarkers in primary biliary cholangitis

IF 0.8 4区 医学 Q4 PHARMACOLOGY & PHARMACY Expert Opinion on Orphan Drugs Pub Date : 2021-04-03 DOI:10.1080/21678707.2021.1927700
G. Mulinacci, A. Palermo, P. Invernizzi, M. Carbone
{"title":"Old and novel prognostic biomarkers in primary biliary cholangitis","authors":"G. Mulinacci, A. Palermo, P. Invernizzi, M. Carbone","doi":"10.1080/21678707.2021.1927700","DOIUrl":null,"url":null,"abstract":"ABSTRACT Introduction: Primary Biliary Cholangitis (PBC) is a chronic liver disease characterized by intrahepatic biliary destruction and progressive cholestasis potentially leading to end-stage liver disease and its associated complications. As for many chronic disorders, the risk of disease progression in PBC varies between patients. This has fostered the identification of disease tags defining the trajectory of the disease course. Areas covered: In this review, authors report the evidence-based parameters associated with outcomes in PBC, after an extensive literature search of the PubMed electronic database. Expert opinion: Several prognostic tools including clinical and biochemical disease parameters have been proposed. In the era of the omics science and digital innovation, biomarker discovery has sped up. Extensive data and sample collection of well-phenotyped cohorts of patients with longitudinal follow-up will guarantee an extraordinary opportunity to integrate molecular phenotyping into the clinic, thus promoting a personalized care of patients with PBC. Finally, we emphasize the need to leverage a therapeutic window of opportunity in PBC bringing a paradigm shift toward early intervention with combination therapies in individuals with a more severe phenotype to reach a deeper disease control and halt disease progression.","PeriodicalId":12118,"journal":{"name":"Expert Opinion on Orphan Drugs","volume":"9 1","pages":"123 - 131"},"PeriodicalIF":0.8000,"publicationDate":"2021-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678707.2021.1927700","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Expert Opinion on Orphan Drugs","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/21678707.2021.1927700","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0

Abstract

ABSTRACT Introduction: Primary Biliary Cholangitis (PBC) is a chronic liver disease characterized by intrahepatic biliary destruction and progressive cholestasis potentially leading to end-stage liver disease and its associated complications. As for many chronic disorders, the risk of disease progression in PBC varies between patients. This has fostered the identification of disease tags defining the trajectory of the disease course. Areas covered: In this review, authors report the evidence-based parameters associated with outcomes in PBC, after an extensive literature search of the PubMed electronic database. Expert opinion: Several prognostic tools including clinical and biochemical disease parameters have been proposed. In the era of the omics science and digital innovation, biomarker discovery has sped up. Extensive data and sample collection of well-phenotyped cohorts of patients with longitudinal follow-up will guarantee an extraordinary opportunity to integrate molecular phenotyping into the clinic, thus promoting a personalized care of patients with PBC. Finally, we emphasize the need to leverage a therapeutic window of opportunity in PBC bringing a paradigm shift toward early intervention with combination therapies in individuals with a more severe phenotype to reach a deeper disease control and halt disease progression.
查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
原发性胆管炎的新旧预后生物标志物
摘要简介:原发性胆管炎(PBC)是一种以肝内胆管破坏和进行性胆汁淤积为特征的慢性肝病,可能导致终末期肝病及其相关并发症。对于许多慢性疾病,PBC的疾病进展风险因患者而异。这促进了确定疾病过程轨迹的疾病标签的识别。涵盖的领域:在这篇综述中,作者在对PubMed电子数据库进行广泛的文献检索后,报告了与PBC结果相关的循证参数。专家意见:已经提出了包括临床和生化疾病参数在内的几种预后工具。在组学科学和数字创新的时代,生物标志物的发现速度加快了。通过纵向随访,对表型良好的患者队列进行广泛的数据和样本收集,将保证将分子表型整合到临床中的非凡机会,从而促进PBC患者的个性化护理。最后,我们强调,需要利用PBC的治疗机会窗口,在表型更严重的个体中,向早期干预和联合治疗转变模式,以达到更深层次的疾病控制和阻止疾病进展。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 去求助
来源期刊
Expert Opinion on Orphan Drugs
Expert Opinion on Orphan Drugs PHARMACOLOGY & PHARMACY-
CiteScore
2.30
自引率
0.00%
发文量
8
期刊介绍: Expert Opinion on Orphan Drugs is an international, peer-reviewed journal that covers all aspects of R&D on rare diseases and orphan drugs.
期刊最新文献
Emerging therapies against Naegleria fowleri Current and emerging therapies for the treatment of leishmaniasis The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-2022 Orphan drugs approved in Canada: health technology assessment, price negotiation, and government formulary listing Overview of genetic testing in Prader-willi syndrome
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1