Department of Dermatology, University of Utah, Salt Lake City.
Division of Rheumatology, Department of Dermatology, Brigham and Women's Hospital Harvard Medical School, Boston, Massachusetts.
JAMA Dermatol. 2018 Oct 1;154(10):1137-1144. doi: 10.1001/jamadermatol.2018.1165.
There is no consensus on which domains should be measured or which instruments should be used in clinical trials for psoriasis therapies.
To achieve international consensus among psoriasis stakeholders on a core set of domains that should be measured in all psoriasis clinical trials.
DESIGN, SETTING, AND PARTICIPANTS: Literature review, pre-Delphi survey exercises, nominal group discussions, and audience voting at 4 stakeholder meetings were used to develop candidate domains for 2 rounds of a Delphi survey. Stakeholders were patients or advocates of patients with psoriasis and health care professionals (HCPs) with expertise in psoriasis, including physicians, scientists, advocacy organization representatives, and regulators. Delphi surveys were conducted electronically from October through December 2015 and between September and October 2016. Stakeholder discussions with audience response voting were conducted at live meetings in the United States, Canada, and Italy from January 2013 to December 2016 to refine and ratify the core set of domains.
Two rounds of an electronic Delphi survey were used to determine consensus. A domain was considered "core" (ie, should be measured in all trials) if a threshold consensus of at least 70% was met in both patient and HCP groups. Domains meeting consensus in only 1 group were considered to be important but were not required to be measured in all trials ("middle ring"). These domains were included for rerating in round 2. Domains that did not meet consensus in either of the groups ("outer ring") were considered to be of uncertain importance and were placed in the research agenda.
In round 1 of the Delphi survey, 107 HCPs and 14 patients participated. Most HCPs (72 [67%]) were dermatologists between 46 and 64 years old (71 [66%]), white (78 [73%]), and male (75 [70%]) from North America (60 [57%]) and Europe (34 [32%]).There were 10 pharmaceutical industry clinical or health economic scientists, 3 advocacy organization representatives, 2 regulatory agency representatives, and 5 "other." In the second round, 77 HCPs and 15 patients participated. Of the 20 candidate domains, the following 6 met consensus as core domains: skin manifestations, psoriasis and psoriatic arthritis symptoms, health-related quality of life, investigator global assessment, patient global assessment, and treatment satisfaction. Secondary skin manifestations as well as nail, inverse, genital, and guttate psoriasis were classified as important but not mandatory. Psoriatic arthritis signs, work productivity or participation, economic impact (direct and indirect cost), and cardiovascular disease comprised the research agenda.
This iterative Delphi process yielded international consensus among professional and patient stakeholders on 6 domains that should be measured in all clinical trials for psoriasis. Future International Dermatology Outcome Measures group efforts will focus on development of a core outcome measurement set for psoriasis trials.
目前在银屑病治疗的临床试验中，对于应该测量哪些领域以及应该使用哪些工具，尚未达成共识。
在银屑病利益相关者中达成共识，确定应在所有银屑病临床试验中测量的核心领域。
设计、设置和参与者：文献回顾、德尔菲调查前练习、名义小组讨论以及在 4 次利益相关者会议上的观众投票，用于开发两轮德尔菲调查的候选领域。利益相关者为银屑病患者或患者的倡导者以及具有银屑病专业知识的医疗保健专业人员（HCP），包括医生、科学家、倡导组织代表和监管机构。德尔菲调查于 2015 年 10 月至 12 月和 2016 年 9 月至 10 月之间以电子方式进行。2013 年 1 月至 2016 年 12 月期间，在美国、加拿大和意大利举行了利益相关者讨论和观众反应投票，以完善和批准核心领域。
两轮电子德尔菲调查用于确定共识。如果患者和 HCP 组都达到至少 70%的阈值共识，则认为该领域是“核心”（即，应在所有试验中进行测量）。仅在 1 组中达成共识的领域被认为是重要的，但不一定需要在所有试验中进行测量（“中环”）。这些领域将在第二轮中重新评估。在这两个组中都没有达成共识的领域（“外环”）被认为重要性不确定，将被列入研究议程。
在第一轮德尔菲调查中，有 107 名 HCP 和 14 名患者参加。大多数 HCP（72 [67%]）为 46 至 64 岁（71 [66%]）、白人（78 [73%]）和男性（75 [70%]），来自北美（60 [57%]）和欧洲（34 [32%]）。有 10 名制药行业临床或健康经济科学家、3 名倡导组织代表、2 名监管机构代表和 5 名“其他”。在第二轮中，有 77 名 HCP 和 15 名患者参加。在 20 个候选领域中，以下 6 个被确认为核心领域：皮肤表现、银屑病和银屑病关节炎症状、健康相关生活质量、研究者总体评估、患者总体评估和治疗满意度。次要皮肤表现以及指甲、反向、生殖器和点滴状银屑病被归类为重要但非强制性的。银屑病关节炎的体征、工作生产力或参与度、经济影响（直接和间接成本）和心血管疾病构成了研究议程。
该迭代德尔菲过程在专业和患者利益相关者中就应在所有银屑病临床试验中测量的 6 个领域达成了共识。未来国际皮肤病学结局测量组的工作重点将是制定银屑病试验的核心结局测量集。
