Department of Rheumatology and Clinical Immunology, University Medical Centre Groningen, The Netherlands.
Clin Exp Rheumatol. 2020 Jul-Aug;38 Suppl 126(4):283-290. Epub 2020 Oct 23.
Primary Sjögren's syndrome (pSS) is a very heterogeneous disease with systemic manifestations such as arthritis, skin, lung and renal involvement. To be able to assess systemic disease activity, the EULAR Sjögren's syndrome disease activity index (ESSDAI) was developed for use in daily clinical practice and in clinical trials. Since its development it has been widely used in cohort studies and clinical trials. The ESSDAI gives a systematic overview of a patient's systemic disease activity, which is very useful in daily clinical practice. However, using the ESSDAI as outcome measure in trials has been more challenging. Several RCTs with the ESSDAI as primary endpoint failed and showed large 'response rates' in placebo-treated patients as well. In this review, we discuss what we learned from using the ESSDAI in cohorts and clinical trials. We recommend to use the ESSDAI only in combination with other important outcome measures, such as patient-reported symptoms and glandular function as part of a composite endpoint in clinical trials in pSS patients.
原发性干燥综合征（pSS）是一种表现非常多样的疾病，可出现关节炎、皮肤、肺部和肾脏等全身表现。为了能够评估全身性疾病活动度，EULAR 干燥综合征疾病活动指数（ESSDAI）被开发出来，用于日常临床实践和临床试验中。自开发以来，它已被广泛用于队列研究和临床试验中。ESSDAI 对患者的全身性疾病活动度进行了系统评估，这在日常临床实践中非常有用。然而，在临床试验中使用 ESSDAI 作为结局指标更具挑战性。几项以 ESSDAI 为主要终点的 RCT 失败了，且安慰剂治疗组的“应答率”也很高。在这篇综述中，我们讨论了从队列研究和临床试验中使用 ESSDAI 中获得的经验。我们建议仅在临床试验中 pSS 患者的复合终点中，将 ESSDAI 与其他重要的结局指标（如患者报告的症状和腺体功能）联合使用。
