用于临床试验和实践的酒渣鼻核心领域集：一项共识声明。

Rosacea Core Domain Set for Clinical Trials and Practice: A Consensus Statement.

作者信息

Dirr McKenzie A, Ahmed Areeba, Schlessinger Daniel I, Haq Misha, Shi Victoria, Koza Eric, Ma Melissa, Christensen Rachel E, Ibrahim Sarah A, Schmitt Jochen, Johannsen Lena, Asai Yuka, Baldwin Hilary E, Berardesca Enzo, Berman Brian, Vieira Ana Carolina, Chien Anna L, Cohen David E, Del Rosso James Q, Dosal Jacquelyn, Drake Lynn A, Feldman Steven R, Fleischer Alan B, Friedman Adam, Graber Emmy, Harper Julie C, Helfrich Yolanda R, Jemec Gregor B, Johnson Sandra M, Katta Rajani, Lio Peter, Maier Lisa E, Martin George, Nagler Arielle R, Neuhaus Isaac M, Palamar Melis, Parish Lawrence C, Rosen Theodore, Shumack Stephen P, Solomon James A, Tanghetti Emil A, Webster Guy F, Weinkle Allison, Weiss Jonathan S, Wladis Edward J, Maher Ian A, Sobanko Joseph F, Cartee Todd V, Cahn Brian A, Alam Murad, Kang Bianca Y, Iyengar Sanjana, Anvery Noor, Alpsoy Erkan, Bewley Anthony, Dessinioti Clio, Egeberg Alexander, Engin Burhan, Gollnick Harald P M, Ioannides Dimitrios, Kim Hei Sung, Lazaridou Elizabeth, Li Ji, Lim Hester Gail, Micali Giuseppe, de Oliveira Clivia Maria Moraes, Noguera-Morel Lucero, Parodi Aurora, Reinholz Markus, Suh Dae Hun, Sun Qiuning, van Zuuren Esther J, Wollina Uwe, Zhou Youwen, Zip Catherine, Poon Emily, Pearlman Ross

机构信息

Department of Dermatology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois.

Center for Evidence-Based Healthcare, Faculty of Medicine Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.

出版信息

JAMA Dermatol. 2024 Jun 1;160(6):658-666. doi: 10.1001/jamadermatol.2024.0636.

DOI:10.1001/jamadermatol.2024.0636

PMID:38656294

Abstract

IMPORTANCE

Inconsistent reporting of outcomes in clinical trials of rosacea is impeding and likely preventing accurate data pooling and meta-analyses. There is a need for standardization of outcomes assessed during intervention trials of rosacea.

OBJECTIVE

To develop a rosacea core outcome set (COS) based on key domains that are globally relevant and applicable to all demographic groups to be used as a minimum list of outcomes for reporting by rosacea clinical trials, and when appropriate, in clinical practice.

EVIDENCE REVIEW

A systematic literature review of rosacea clinical trials was conducted. Discrete outcomes were extracted and augmented through discussions and focus groups with key stakeholders. The initial list of 192 outcomes was refined to identify 50 unique outcomes that were rated through the Delphi process Round 1 by 88 panelists (63 physicians from 17 countries and 25 patients with rosacea in the US) on 9-point Likert scale. Based on feedback, an additional 11 outcomes were added in Round 2. Outcomes deemed to be critical for inclusion (rated 7-9 by ≥70% of both groups) were discussed in consensus meetings. The outcomes deemed to be most important for inclusion by at least 85% of the participants were incorporated into the final core domain set.

FINDINGS

The Delphi process and consensus-building meetings identified a final core set of 8 domains for rosacea clinical trials: ocular signs and symptoms; skin signs of disease; skin symptoms; overall severity; patient satisfaction; quality of life; degree of improvement; and presence and severity of treatment-related adverse events. Recommendations were also made for application in the clinical setting.

CONCLUSIONS AND RELEVANCE

This core domain set for rosacea research is now available; its adoption by researchers may improve the usefulness of future trials of rosacea therapies by enabling meta-analyses and other comparisons across studies. This core domain set may also be useful in clinical practice.

摘要

重要性

酒渣鼻临床试验中结果报告的不一致阻碍且可能妨碍了准确的数据汇总和荟萃分析。在酒渣鼻干预试验期间评估的结果需要标准化。

目的

基于全球相关且适用于所有人口群体的关键领域，制定一个酒渣鼻核心结局集（COS），作为酒渣鼻临床试验报告的最低结局清单，并在适当情况下用于临床实践。

证据审查

对酒渣鼻临床试验进行了系统的文献综述。通过与关键利益相关者的讨论和焦点小组提取并扩充了离散的结局。最初的192个结局清单经过提炼，确定了50个独特的结局，88名小组成员（来自17个国家的63名医生和美国的25名酒渣鼻患者）在第一轮德尔菲过程中以9分制李克特量表对其进行了评分。根据反馈，在第二轮中又增加了11个结局。在共识会议上讨论了被认为对纳入至关重要的结局（两组中≥70%的人评分为7 - 9分）。至少85%的参与者认为对纳入最重要的结局被纳入最终的核心领域集。