Lu J D, Hobbs M M, Huang W W, Ortega-Loayza A G, Alavi A
Michael G. DeGroote School of Medicine, McMaster University, Hamilton, ON, Canada.
University of Kentucky College of Medicine, Lexington, KY, USA.
Br J Dermatol. 2020 Nov;183(5):821-828. doi: 10.1111/bjd.19027. Epub 2020 May 7.
Pyoderma gangrenosum (PG) is a rare autoinflammatory skin condition that causes tissue destruction and subsequent painful ulcers. To date, there are no core domains or instruments for assessing PG severity in clinical trials, and current treatment paradigms rely on outcome measurements that have not been well characterized in the literature.
To perform two systematic reviews that (i) identify the outcome measurement instruments used in PG clinical trials and their corresponding domains and (ii) identify any associated validation studies and evaluate their measurement properties and methodological quality.
We systematically searched the MEDLINE and Embase databases for PG outcome measurement instruments. We also systematically searched for PG instrument validation studies. We evaluated the measurement properties and methodological quality of validation studies using the 2018 COSMIN Risk of Bias checklist.
In total, seven clinical trials were included. These studies utilized a total of 20 different instruments, including 11 physician-reported instruments, eight patient-reported instruments and one composite instrument. Among these, 85% of the instruments lacked any validation data. Of the remaining three validated instruments (speed of healing, physician global assessment and resolution of inflammation), methodological quality was not available for half of the COSMIN categories.
We identified 17 non-validated outcome measurement instruments used in PG clinical trials. We conclude that PG validation studies are required for existing instruments, and new instruments need to be developed to inform the consensus process for the development of a core outcome set for PG. What is already known about this topic? Pyoderma gangrenosum (PG) is a rare autoinflammatory skin condition that has been characterized by multiple outcome measurement instruments in clinical trials. However, there is no consensus on the most validated and appropriate outcome measurement instruments. What does this study add? This study identifies and evaluates 20 unique outcome measurement instruments for PG in the literature. Of these 20, 17 lack any instrument validation data, highlighting the need for future studies. What are the clinical implications of this work? Despite the current use of several outcome measurement instruments, future studies should explore the validation surrounding these instruments, as no instruments can currently be recommended.
坏疽性脓皮病（PG）是一种罕见的自身炎症性皮肤病，可导致组织破坏并继而形成疼痛性溃疡。迄今为止，在临床试验中尚无用于评估PG严重程度的核心领域或工具，当前的治疗模式依赖于文献中尚未充分描述的结局测量指标。
进行两项系统评价，（i）确定PG临床试验中使用的结局测量工具及其相应领域，（ii）确定任何相关的验证研究，并评估其测量特性和方法学质量。
我们系统检索MEDLINE和Embase数据库以查找PG结局测量工具。我们还系统检索了PG工具验证研究。我们使用2018年COSMIN偏倚风险清单评估验证研究的测量特性和方法学质量。
总共纳入了7项临床试验。这些研究共使用了20种不同的工具，包括11种医生报告的工具、8种患者报告的工具和1种综合工具。其中，85%的工具缺乏任何验证数据。在其余3种经过验证的工具（愈合速度、医生整体评估和炎症消退）中，一半的COSMIN类别没有方法学质量数据。
我们确定了PG临床试验中使用的17种未经验证的结局测量工具。我们得出结论，现有工具需要进行PG验证研究，并且需要开发新的工具，以为PG核心结局集的制定提供共识依据。关于该主题已知的内容有哪些？坏疽性脓皮病（PG）是一种罕见的自身炎症性皮肤病，在临床试验中已有多种结局测量工具对其进行了描述。然而，对于最经过验证且合适的结局测量工具尚无共识。本研究增加了什么内容？本研究在文献中识别并评估了20种用于PG的独特结局测量工具。在这20种工具中，17种缺乏任何工具验证数据，凸显了未来研究的必要性。这项工作的临床意义是什么？尽管目前使用了多种结局测量工具，但未来的研究应探索围绕这些工具的验证情况，因为目前尚无工具可被推荐。
