Khanna D, Lovell D J, Giannini E, Clements P J, Merkel P A, Seibold J R, Matucci-Cerinic M, Denton C P, Mayes M D, Steen V D, Varga J, Furst D E
Division of Rheumatology, Department of Medicine, David Geffen School of Medicine, University of California at Los Angeles, California 90095, USA.
Ann Rheum Dis. 2008 May;67(5):703-9. doi: 10.1136/ard.2007.078923. Epub 2007 Sep 24.
To develop a provisional core set of response measures for clinical trials of systemic sclerosis (SSc).
The Scleroderma Clinical Trials Consortium (SCTC) conducted a structured, 3-round Delphi exercise to reach consensus on a core set of measures for clinical trials of SSc. Round 1 asked the SCTC investigators to list items in 11 pre-defined domains (skin, musculoskeletal, cardiac, pulmonary, cardio-pulmonary, gastrointestinal, renal, Raynaud phenomenon and digital ulcers, health-related quality of life and function, global health, and biomarkers) for SSc clinical trials. Round 2 asked respondents to rate the importance of the chosen items and was followed by a meeting, during which the Steering Committee discussed the feasibility, reliability, redundancy and validity of the items. Round 3 sought to obtain broader consensus on the core set measures. Members also voted on items that had data on feasibility but lacked data on reliability and validity, but may still be useful research outcome measures for future trials.
A total of 50 SCTC investigators participated in round 1, providing 212 unique items for the 11 domains. In all, 46 (92%) participants responded in round 2 and rated 177 items. The ratings of 177 items were reviewed by the Steering Committee and 31 items from the 11 domains were judged to be appropriate for inclusion in a 1-year multi-centre clinical trial. In total, 40 SCTC investigators completed round 3 and ranked 30 of 31 items as acceptable for inclusion in the core set. The Steering Committee also proposed 14 items for a research agenda.
Using a Delphi exercise, we have developed a provisional core set of measures for assessment of disease activity and severity in clinical trials of SSc.
制定系统性硬化症（SSc）临床试验的一套临时核心反应指标。
硬皮病临床试验联盟（SCTC）开展了一项结构化的三轮德尔菲法练习，以就SSc临床试验的一套核心指标达成共识。第一轮要求SCTC研究人员列出SSc临床试验11个预定义领域（皮肤、肌肉骨骼、心脏、肺、心肺、胃肠道、肾脏、雷诺现象和指端溃疡、健康相关生活质量和功能、整体健康以及生物标志物）中的项目。第二轮要求受访者对所选项目的重要性进行评分，随后召开了一次会议，指导委员会在会上讨论了这些项目的可行性、可靠性、冗余性和有效性。第三轮旨在就核心指标达成更广泛的共识。成员们还对那些有可行性数据但缺乏可靠性和有效性数据，但可能仍是未来试验有用的研究结果指标的项目进行了投票。
共有50名SCTC研究人员参与了第一轮，为11个领域提供了212个独特项目。总共有46名（92%）参与者参与了第二轮并对177个项目进行了评分。指导委员会对177个项目的评分进行了审查，11个领域中的31个项目被判定适合纳入为期1年的多中心临床试验。共有40名SCTC研究人员完成了第三轮，将31个项目中的30个列为可纳入核心指标集。指导委员会还为一项研究议程提出了14个项目。
通过德尔菲法练习，我们制定了一套临时核心指标，用于评估SSc临床试验中的疾病活动度和严重程度。
