Connelly Kathryn, Golder Vera, Kandane-Rathnayake Rangi, Morand Eric F
School of Clinical Sciences, Monash University, Clayton, VIC 3168, Australia; Department of Rheumatology, Monash Health, Monash University, Clayton, VIC 3168, Australia.
School of Clinical Sciences, Monash University, Clayton, VIC 3168, Australia.
Lancet Rheumatol. 2021 Aug;3(8):e595-e603. doi: 10.1016/S2665-9913(21)00119-3. Epub 2021 Jun 7.
Systemic lupus erythematosus (SLE) remains a disease of high unmet clinical need. Because of substantial patient heterogeneity, the execution of clinical trials that successfully determine the efficacy of novel therapeutics compared with placebo is a continuous challenge. Clinician-reported outcome measures of treatment response used in SLE trials have evolved from the use of individual disease activity indices, including the SLE Disease Activity Index (SLEDAI) and British Isles Lupus Assessment Group (BILAG), to composite responder definitions such as the SLE Responder Index (SRI) and BILAG-Based Composite Lupus Assessment (BICLA), which are based on these indices. However, these approaches have notable drawbacks and defining the optimal clinical trial outcome measure for SLE remains a research goal. In this Viewpoint, we explore the strengths and limitations of existing indices and composite assessments, illustrating features which should be investigated in future analysis of trial data. Further, we provide a platform from which to advance new approaches to endpoint design, which is crucial to improve the interpretability and success of subsequent clinical trials in SLE.
系统性红斑狼疮(SLE)仍然是一种临床需求远未满足的疾病。由于患者存在显著的异质性,开展与安慰剂相比能成功确定新型疗法疗效的临床试验一直是一项挑战。SLE试验中使用的临床医生报告的治疗反应结局指标已从使用个体疾病活动指数(包括SLE疾病活动指数(SLEDAI)和英伦三岛狼疮评估组(BILAG))发展到基于这些指数的综合反应定义,如SLE反应指数(SRI)和基于BILAG的综合狼疮评估(BICLA)。然而,这些方法存在显著缺陷,确定SLE的最佳临床试验结局指标仍然是一个研究目标。在本观点文章中,我们探讨了现有指数和综合评估的优势与局限性,阐述了在未来试验数据分析中应研究的特征。此外,我们提供了一个推进终点设计新方法的平台,这对于提高后续SLE临床试验的可解释性和成功率至关重要。