Department of Rheumatology, Leiden University Medical Center, PO Box 9600, 2300 RC Leiden, The Netherlands.
Joint Bone Spine. 2023 Jul;90(4):105543. doi: 10.1016/j.jbspin.2023.105543. Epub 2023 Feb 15.
During the last decade, the outlook for patients with rheumatoid arthritis (RA) has improved greatly, especially for patients with autoantibody-positive RA. To further improve long-term disease outcomes, the field has turned to investigating the efficacy of treatment initiated in the pre-arthritic phase of RA, based on the adage "the sooner the better." In this review, the concept of prevention is evaluated and different risk stages are being examined for their pre-test risks of RA development. These risks affect the post-test risk of biomarkers used at these stages and, consequently, the accuracy with which the risk of RA can be estimated. Furthermore, through their effect on accurate risk stratification, these pre-test risks ultimately also associate with the likelihood of false-negative trial results (the so-called "clinicostatistical tragedy"). Outcome measures to assess preventive effects are evaluated and relate to either the occurrence of disease itself or to the severity of risk factors for RA development. Results of recently completed prevention studies are discussed in the light of these theoretical considerations. The results vary, but clear prevention of RA has not been demonstrated. While some treatments (e.g. methotrexate) persistently reduced symptom severity, physical disability, and the severity of imaging joint inflammation, other treatments were not reported to produce long-lasting effects (hydroxychloroquine, rituximab, atorvastatin). The review concludes with future perspectives regarding the design of new prevention studies and considerations and requirements before findings can be implemented in daily practice in individuals at risk of RA attending rheumatology practices.
在过去的十年中，类风湿关节炎（RA）患者的前景有了很大改善，尤其是对于自身抗体阳性的 RA 患者。为了进一步改善长期疾病结局，该领域已转向研究在 RA 的关节炎前期开始治疗的疗效，这一理念基于“越早越好”。在这篇综述中，评估了预防的概念，并检查了不同的风险阶段，以评估其发生 RA 的预先测试风险。这些风险影响这些阶段使用的生物标志物的后测试风险，从而影响对 RA 风险的估计的准确性。此外，通过对准确的风险分层的影响，这些预先测试风险最终也与假阴性试验结果（所谓的“临床统计学悲剧”）的可能性相关。评估了用于评估预防效果的结果测量指标，这些测量指标与疾病本身的发生或 RA 发展的危险因素的严重程度有关。根据这些理论考虑，讨论了最近完成的预防研究的结果。结果各不相同，但尚未证明明确的预防 RA 的效果。虽然一些治疗方法（例如甲氨蝶呤）持续降低了症状严重程度、身体残疾和影像学关节炎症的严重程度，但其他治疗方法并未报告产生持久效果（羟氯喹、利妥昔单抗、阿托伐他汀）。该综述以新的预防研究设计的未来展望以及在风险人群中实施之前的考虑因素和要求结束，这些人群在接受风湿病学治疗的风险人群中易患 RA。
