银屑病关节炎临床试验的综合指标：在一项英国多中心研究中检验疼痛和疲劳的改变。

Composite Measures for Clinical Trials in Psoriatic Arthritis: Testing Pain and Fatigue Modifications in a UK Multicenter Study.

机构信息

W. Tillett, BSc, MBChB, PhD, FRCP, N.J. McHugh, MBChB, MD, FRCP, FRCPath, Department of Pharmacy and Pharmacology, University of Bath, and Royal National Hospital for Rheumatic Diseases, Bath, UK;

O. FitzGerald, MD, FRCPI, FRCP, Conway Institute for Biomolecular Research, University College Dublin, Dublin, Ireland.

出版信息

J Rheumatol Suppl. 2021 Jun;97:39-44. doi: 10.3899/jrheum.201674.

DOI:10.3899/jrheum.201674

PMID:34074665

Abstract

OBJECTIVE

To test the addition of pain and fatigue to the Composite Psoriatic Arthritis Disease Activity (CPDAI) and the Group for Research and Assessment of Psoriasis and PsA (GRAPPA) Composite Exercise (GRACE) composite measures of psoriatic arthritis (PsA).

METHODS

Clinical and patient-reported outcome measures were assessed in patients with PsA at 3 consecutive follow-up visits over 6 months in a UK multicenter observational study. A pain visual analog scale and Functional Assessment of Chronic Illness Therapy Fatigue scale were added as modifications to the CPDAI and GRACE composite measures. Original and modified versions were tested against the PsA Disease Activity Score (PASDAS) and the Disease Activity Index for PsA (DAPSA). Discrimination between disease states and responsiveness were tested with -scores, standardized response means (SRMs), and effect sizes. Data were presented to members at the 2020 annual meeting who then voted on the GRAPPA-recommended composite and treatment targets for clinical trials.

RESULTS

One hundred forty-one patients were recruited with a mean PsA disease duration of 6.1 years (range 0-41 yrs). The SRMs for the GRACE and modified GRACE (mGRACE) were 0.67 and 0.64, respectively, and 0.54 and 0.46, respectively, for the CPDAI and modified CPDAI (mCPDAI). The -scores for the GRACE and mGRACE were unchanged at 7.8 for both, and 6.8 and 7.0 for the CPDAI and mCPDAI, respectively. The PASDAS demonstrated the best responsiveness (SRM 0.84) and discrimination (-scores 8.3). Most members (82%) agreed the composites should not be modified and 77% voted for the PASDAS as the GRAPPA-recommended composite for clinical trials, with 90% minimal disease activity (MDA) as the target.

CONCLUSION

Modifying the CPDAI and GRACE with the addition of pain and fatigue does not enhance responsiveness nor the measures' ability to detect disease status in terms of requiring treatment escalation. GRAPPA members voted for the PASDAS as the composite measure in clinical trials and MDA as the target.

摘要

目的

测试疼痛和疲劳对复合银屑病关节炎疾病活动度（CPDAI）和银屑病关节炎研究与评估组（GRAPPA）复合运动（GRACE）复合测量的影响。

方法

在英国多中心观察性研究中，对连续 6 个月的 3 次随访中患有银屑病关节炎的患者进行临床和患者报告的结果测量。疼痛视觉模拟量表和慢性病治疗疲劳量表功能评估被添加到 CPDAI 和 GRACE 综合测量中作为修改。原始和修改版本与银屑病关节炎疾病活动评分（PASDAS）和银屑病关节炎疾病活动指数（DAPSA）进行了对比。使用 -分数、标准化反应均值（SRM）和效应大小测试疾病状态的区分度和反应性。数据提交给 2020 年年会的成员，然后成员投票决定 GRAPPA 推荐的临床试验综合测量和治疗目标。

结果

共招募了 141 名患者，平均银屑病关节炎病程为 6.1 年（范围 0-41 年）。GRACE 和改良 GRACE（mGRACE）的 SRM 分别为 0.67 和 0.64，CPDAI 和改良 CPDAI（mCPDAI）的 SRM 分别为 0.54 和 0.46。GRACE 和 mGRACE 的 -分数分别为 7.8，CPDAI 和 mCPDAI 的 -分数分别为 6.8 和 7.0。PASDAS 显示出最佳的反应性（SRM 0.84）和区分度（-分数 8.3）。大多数成员（82%）同意这些综合测量不应修改，77%的成员投票赞成 PASDAS 作为临床试验的 GRAPPA 推荐综合测量，90%的最小疾病活动（MDA）作为目标。