Section of Musculoskeletal Disease, Leeds Institute of Molecular Medicine, University of Leeds, 2nd Floor, Chapel Allerton Hospital, Chapeltown Road, Leeds LS7 4SA, UK.
Ann Rheum Dis. 2011 Jun;70(6):886-90. doi: 10.1136/ard.2010.143420. Epub 2011 Mar 6.
Double-blind, randomised controlled studies represent the gold-standard approach to determine the safety and efficacy of therapeutic interventions. In chronic conditions such as rheumatoid arthritis (RA), long-term data are vital to confirm maintenance of effect and identify potential safety signals. The recent introduction of numerous biological therapies for RA has been followed by various long-term extension (LTE) studies. Although useful, the design and method of analysis in such studies vary significantly, partly due to their complexity. This viewpoint highlights general considerations needed when undertaking a LTE study and illustrates the lack of consistency in studies of RA to date. It addresses issues of selection bias, patient discontinuation and missing data. Although used for safety reporting, the lack of adequate powering makes LTE studies of limited benefit. Ethical considerations and challenges are highlighted, including potential conflicts of interest. Finally, the authors suggest the need for consensus to ensure more reliable interpretation and application of data for clinical practice. Following the development of guidelines on reporting of clinical trials in RA and more recently, registry data, a similar approach for LTE studies would be a useful endeavour.
双盲、随机对照研究是确定治疗干预安全性和疗效的金标准方法。在类风湿关节炎（RA）等慢性疾病中，长期数据对于确认疗效的维持和识别潜在的安全信号至关重要。最近，针对 RA 的多种生物疗法相继推出了各种长期扩展（LTE）研究。虽然这些研究很有用，但由于其复杂性，它们的设计和分析方法存在很大差异。本观点强调了进行 LTE 研究时需要考虑的一般因素，并说明了迄今为止 RA 研究中缺乏一致性的情况。它涉及选择偏差、患者停药和缺失数据等问题。尽管 LTE 研究用于安全性报告，但由于缺乏足够的效力，其益处有限。本文还强调了伦理考虑和挑战，包括潜在的利益冲突。最后，作者建议需要达成共识，以确保更可靠地解释和应用数据用于临床实践。在 RA 临床试验报告指南的制定以及最近的登记数据之后，针对 LTE 研究采用类似的方法将是一项有益的努力。
