在两项原发性干燥综合征随机对照试验中，由频繁活跃的临床领域组成的临床试验ESSDAI（ClinTrialsESSDAI）的开发与性能。
Development and performance of the Clinical Trials ESSDAI (ClinTrialsESSDAI), consisting of frequently active clinical domains, in two randomised controlled trials in primary Sjögren's syndrome.
作者信息
de Wolff Liseth, Arends Suzanne, Pontarini Elena, Bombardieri Michele, Bowman Simon J, Bootsma Hendrika
机构信息
Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Centre Groningen, the Netherlands.
Queen Mary University of London, William Harvey Research Institute, Centre for Experimental Medicine and Rheumatology, London, UK.
出版信息
Clin Exp Rheumatol. 2021 Nov-Dec;39 Suppl 133(6):100-106. doi: 10.55563/clinexprheumatol/i8g5nd. Epub 2021 Nov 10.
OBJECTIVES
To develop and evaluate the Clinical Trials EULAR Sjögren's Syndrome Disease Activity Index (ClinTrialsESSDAI), consisting of frequently active clinical domains of the ESSDAI, using two randomised controlled trials in primary Sjögren's syndrome (pSS).
METHODS
The ASAP-III trial in abatacept (80 pSS patients) and TRACTISS trial in rituximab (133 pSS patients) were analysed. The most frequently active clinical domains were selected, and ClinTrialsESSDAI total score was calculated using existing weightings of the ClinESSDAI (which also excludes the biological domain). Performance of the ClinTrialsESSDAI was compared to ClinESSDAI and ESSDAI. Responsiveness was assessed using standardised response mean (SRM), and discrimination was assessed using adjusted mean difference.
RESULTS
Besides the biological domain, the most frequently active domains were glandular, articular, haematological, constitutional, lymphadenopathy and cutaneous. These domains were selected for the ClinTrialsESSDAI. At primary endpoint visits, SRM values of ClinTrialsESSDAI, ClinESSDAI and ESSDAI were respectively -0.65/-0.59, -0.63/-0.59 and -0.64/-0.61 for abatacept/placebo and -0.33/-0.13, -0.34/-0.12 and -0.41/-0.16 for rituximab/placebo. Adjusted mean differences between active treatment and placebo groups were respectively -1.7, -1.4 and -1.1 for ASAP-III and -1.1, -1.1 and -1.2 for TRACTISS.
CONCLUSIONS
The ClinTrialsESSDAI, consisting of six frequently active clinical domains of the ESSDAI, shows closely similar responsiveness and discrimination between treatment groups compared to the ClinESSDAI and ESSDAI. Therefore, this ClinTrialsESSDAI is not preferable to ClinESSDAI and ESSDAI for use as primary endpoint. A composite endpoint combining response at multiple clinically relevant items seems more suitable as primary study endpoint in pSS.
目的
利用两项原发性干燥综合征（pSS）的随机对照试验，开发并评估由欧洲抗风湿病联盟干燥综合征疾病活动指数（ESSDAI）中频繁出现活动的临床领域组成的临床试验ESSDAI（ClinTrialsESSDAI）。
方法
分析了阿巴西普的ASAP-III试验（80例pSS患者）和利妥昔单抗的TRACTISS试验（133例pSS患者）。选择最常出现活动的临床领域，并使用ClinESSDAI的现有权重（也排除生物学领域）计算ClinTrialsESSDAI总分。将ClinTrialsESSDAI的表现与ClinESSDAI和ESSDAI进行比较。使用标准化反应均值（SRM）评估反应性，并使用调整后的均值差异评估区分度。
结果
除生物学领域外，最常出现活动的领域是腺体、关节、血液学、全身症状、淋巴结病和皮肤。这些领域被选入ClinTrialsESSDAI。在主要终点访视时，阿巴西普/安慰剂组的ClinTrialsESSDAI、ClinESSDAI和ESSDAI的SRM值分别为-0.65/-0.59、-0.63/-0.59和-0.64/-0.61，利妥昔单抗/安慰剂组分别为-0.33/-0.13、-0.34/-0.12和-0.41/-0.16。ASAP-III试验中活性治疗组与安慰剂组的调整后均值差异分别为-1.7、-1.4和-1.1，TRACTISS试验分别为-1.1、-1.1和-1.2。
结论
由ESSDAI的六个频繁出现活动的临床领域组成的ClinTrialsESSDAI，与ClinESSDAI和ESSDAI相比，在治疗组之间显示出非常相似的反应性和区分度。因此，作为主要终点，ClinTrialsESSDAI并不比ClinESSDAI和ESSDAI更可取。结合多个临床相关项目反应的复合终点似乎更适合作为pSS的主要研究终点。
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