医学
疾病
临床终点
重症监护医学
全身性疾病
临床试验
干燥综合征
淋巴瘤
内科学
梅德林
美罗华
疾病严重程度
全身疗法
免疫学
作者
Maxime Beydon,Simon J Bowman,Raphaèle Séror
标识
DOI:10.1016/j.coi.2026.102741
摘要
Outcomes in Sjögren disease (SjD) have ranged from diverse symptom scales to the validated EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI) and EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI), which now provide a common framework for evaluating symptoms and systemic activity. In this review, we trace the evolution of trial endpoints and highlight that the first positive trials used the ESSDAI as the primary endpoint. We examine how systemic activity does not necessarily correlate with patient-reported outcomes (PROs), and stress that both dimensions must be evaluated to capture the full spectrum of SjD. New tools are emerging, such as the Sjögren's Tool for Assessing Response (STAR), a composite endpoint requiring improvement in both systemic activity and either PROs or objective sicca measures. Although still recent, STAR could be refined, as current thresholds for ESSDAI and ESSPRI, which were validated as clinically meaningful, could contribute to a substantial placebo effect. Moreover, we argue that future developments should also address organ damage, flares, histological response, and the link between outcome measures and major events such as lymphoma or mortality.
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