Monica T Hannani, Christian S Thudium, Morten A Karsdal, Christoph Ladel, Ali Mobasheri, Melanie Uebelhoer, Jonathan Larkin, Jaume Bacardit, André Struglics, Anne-Christine Bay-Jensen
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引用次数: 0
摘要
导言:骨关节炎(OA)影响着全球 5 亿多人。骨关节炎患者主要接受对症治疗,目前的疗法疗效甚微,而且长期使用往往存在安全隐患。迄今为止,还没有任何改变病情的疗法获得批准,只能将关节置换手术作为最后的手段。为了对患者进行有效治疗并成功开发药物,迫切需要揭示 OA 的病理生物学驱动因素以及这些因素如何转化为疾病内型。与可观察到的表型相比,内型为疾病亚群提供了更精确、更符合机理的定义,而组织和病理特异性生化标记物可能会发现可治疗的 OA 内型:我们在PubMed上搜索了用英文撰写的全文文章,对用于OA内分型的一系列经过验证的生化标记物及其与主要OA病理的关联进行了深入的叙述性综述:正如IMI-APPROACH所采用并经OAI-FNIH验证的那样,一组生化标记物可发现疾病亚群,并有助于丰富可治疗的分子内型,以便纳入治疗性临床试验。了解生化标志物与患者报告结果之间的联系,以及可能对特定疗法产生反应的可治疗内型,将为开发治疗 OA 的新药铺平道路。
From biochemical markers to molecular endotypes of osteoarthritis: a review on validated biomarkers.
Introduction: Osteoarthritis (OA) affects over 500 million people worldwide. OA patients are symptomatically treated, and current therapies exhibit marginal efficacy and frequently carry safety-risks associated with chronic use. No disease-modifying therapies have been approved to date leaving surgical joint replacement as a last resort. To enable effective patient care and successful drug development there is an urgent need to uncover the pathobiological drivers of OA and how these translate into disease endotypes. Endotypes provide a more precise and mechanistic definition of disease subgroups than observable phenotypes, and a panel of tissue- and pathology-specific biochemical markers may uncover treatable endotypes of OA.
Areas covered: We have searched PubMed for full-text articles written in English to provide an in-depth narrative review of a panel of validated biochemical markers utilized for endotyping of OA and their association to key OA pathologies.
Expert opinion: As utilized in IMI-APPROACH and validated in OAI-FNIH, a panel of biochemical markers may uncover disease subgroups and facilitate the enrichment of treatable molecular endotypes for recruitment in therapeutic clinical trials. Understanding the link between biochemical markers and patient-reported outcomes and treatable endotypes that may respond to given therapies will pave the way for new drug development in OA.
期刊介绍:
Expert Review of Molecular Diagnostics (ISSN 1473-7159) publishes expert reviews of the latest advancements in the field of molecular diagnostics including the detection and monitoring of the molecular causes of disease that are being translated into groundbreaking diagnostic and prognostic technologies to be used in the clinical diagnostic setting.
Each issue of Expert Review of Molecular Diagnostics contains leading reviews on current and emerging topics relating to molecular diagnostics, subject to a rigorous peer review process; editorials discussing contentious issues in the field; diagnostic profiles featuring independent, expert evaluations of diagnostic tests; meeting reports of recent molecular diagnostics conferences and key paper evaluations featuring assessments of significant, recently published articles from specialists in molecular diagnostic therapy.
Expert Review of Molecular Diagnostics provides the forum for reporting the critical advances being made in this ever-expanding field, as well as the major challenges ahead in their clinical implementation. The journal delivers this information in concise, at-a-glance article formats: invaluable to a time-constrained community.