Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
Musculoskeletal Unit, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Nat Rev Rheumatol. 2022 Dec;18(12):725-733. doi: 10.1038/s41584-022-00845-w. Epub 2022 Oct 10.
For many oncological conditions, the application of timely and patient-tailored targeted therapies, or precision medicine, is a major therapeutic development that has provided considerable clinical benefit. However, despite the application of increasingly sophisticated technologies, alongside advanced bioinformatic and machine-learning algorithms, this success is yet to be replicated for the rheumatic diseases. In rheumatoid arthritis, for example, despite an array of targeted biologic and conventional therapeutics, treatment choice remains largely based on trial and error. The concept of the 'precision gap' for rheumatic disease can help us to identify factors that underpin the slow progress towards the discovery and adoption of precision-medicine approaches for rheumatic disease. In a rheumatic disease such as rheumatoid arthritis, it is possible to identify four themes that have slowed progress, solutions to which should help to close the precision gap. These themes relate to our fundamental understanding of disease pathogenesis, how we determine treatment response, confounders of treatment outcomes and trial design.
对于许多肿瘤疾病，及时应用针对患者个体的靶向治疗，即精准医学，是一项重大的治疗进展，为患者带来了显著的临床获益。然而，尽管应用了越来越复杂的技术，并结合了先进的生物信息学和机器学习算法，这种成功在风湿性疾病中尚未得到复制。例如，在类风湿关节炎中，尽管有一系列靶向生物制剂和常规治疗药物，治疗选择仍然主要基于反复尝试。精准医学在风湿性疾病中存在“差距”这一概念有助于我们识别出影响其向精准医学方法发现和应用缓慢进展的因素。在类风湿关节炎等风湿性疾病中，可以确定四个阻碍进展的主题，解决这些主题有助于缩小精准医学差距。这些主题与我们对疾病发病机制的基本认识、我们如何确定治疗反应、治疗结果的混杂因素以及临床试验设计有关。
