Mucous Membrane Pemphigoid Outcome Measurement in Clinical Research: A Scoping Review.

IF 2.7 3区 医学 Q2 PHARMACOLOGY & PHARMACY
Billal Tedbirt, Eva W H Korte, Marjolein A J Hiel, Joost M Meijer, Maria C Bolling, Phyllis I Spuls, Sjoukje van der Werf, Marc Yale, Barbara Horváth, Pascal Joly
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Abstract

Background: Mucous membrane pemphigoid (MMP) is a rare autoimmune bullous disease that predominantly affects mucosae. Treatments for MMP often lack robust evidence and may be poorly tolerated, especially in elderly patients.

Objective: This scoping review aims to provide an overview of MMP outcome measurement of the last two decades by mapping and listing all previously reported outcomes and outcome measurement instruments (OMIs).

Design: A large scoping review was performed in scientific databases and trial registries (MEDLINE, Embase, CINAHL, PsycINFO, Cochrane CENTRAL, Web of Science, the International Clinical Trials Registry Platform and Clinicaltrials.gov) covering January 2002 to December 2023. Clinical trials, prospective cohort studies, and systematic reviews were included, while retrospective studies were excluded.

Results: Thirty-nine studies met the inclusion criteria, including 20 prospective cohort studies, 13 systematic reviews, and 6 clinical trials. A total of 285 outcomes were extracted verbatim and categorized into 29 domains and 9 overarching areas. The most commonly reported outcome areas were related to clinical response, safety, and resource use. The majority were investigator-reported outcomes (85%), with only 8% being patient-reported. Various OMIs (n = 14) were employed, including the Mucous Membrane Pemphigoid Disease Area Index (MMPDAI) score and tools for assessing ocular impairment and quality of life. Reported outcomes and OMIs are very diverse due to the heterogeneity in the presentation of MMP.

Conclusions: The standardized use of consensus-based outcome definitions and high-quality instruments that consider the patient perspective is expected to increase the efficacy of future MMP research and can be achieved by consensus-based selection of outcomes and critical appraisal of the measurement properties of OMIs.

临床研究中的粘膜类天疱疮结局测量:范围综述。
背景:粘膜类天疱疮(MMP)是一种罕见的自身免疫性大疱性疾病,主要影响粘膜。MMP的治疗通常缺乏有力的证据,并且可能耐受性差,特别是在老年患者中。目的:本范围综述旨在通过绘制和列出所有先前报告的结果和结果测量工具(OMIs),概述过去二十年的MMP结果测量。设计:对2002年1月至2023年12月的科学数据库和试验注册(MEDLINE、Embase、CINAHL、PsycINFO、Cochrane CENTRAL、Web of Science、国际临床试验注册平台和Clinicaltrials.gov)进行了大范围的综述。纳入临床试验、前瞻性队列研究和系统评价,排除回顾性研究。结果:39项研究符合纳入标准,包括20项前瞻性队列研究、13项系统评价和6项临床试验。我们逐字提取了285个结果,并将其分为29个领域和9个总体领域。最常报道的结果领域与临床反应、安全性和资源利用有关。大多数是研究者报告的结果(85%),只有8%是患者报告的结果。采用各种OMIs (n = 14),包括粘膜类天疱疮疾病面积指数(MMPDAI)评分和评估眼部损害和生活质量的工具。由于MMP表现的异质性,报告的结果和omi非常多样化。结论:标准化使用基于共识的结局定义和考虑患者观点的高质量工具有望提高未来MMP研究的有效性,并且可以通过基于共识的结局选择和对OMIs测量特性的批判性评估来实现。
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来源期刊
CiteScore
5.90
自引率
3.10%
发文量
108
审稿时长
6-12 weeks
期刊介绍: Clinical Drug Investigation provides rapid publication of original research covering all phases of clinical drug development and therapeutic use of drugs. The Journal includes: -Clinical trials, outcomes research, clinical pharmacoeconomic studies and pharmacoepidemiology studies with a strong link to optimum prescribing practice for a drug or group of drugs. -Clinical pharmacodynamic and clinical pharmacokinetic studies with a strong link to clinical practice. -Pharmacodynamic and pharmacokinetic studies in healthy volunteers in which significant implications for clinical prescribing are discussed. -Studies focusing on the application of drug delivery technology in healthcare. -Short communications and case study reports that meet the above criteria will also be considered. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Clinical Drug Investigation may be accompanied by plain language summaries to assist readers who have some knowledge, but non in-depth expertise in, the area to understand important medical advances.
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