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

Area of Science:

• Autoimmune bullous diseases
• Dermatology
• Clinical outcomes research

Background:

• Mucous membrane pemphigoid (MMP) is a rare autoimmune disease affecting mucous membranes.
• Current MMP treatments lack strong evidence and can be poorly tolerated, particularly in elderly patients.

Purpose of the Study:

• To comprehensively map and list all reported outcomes and outcome measurement instruments (OMIs) for MMP over the past two decades.
• To provide an overview of MMP outcome measurement in scientific literature.

Main Methods:

• A broad scoping review was conducted across major scientific databases and clinical trial registries.
• Included studies spanned January 2002 to December 2023, focusing on clinical trials, prospective cohort studies, and systematic reviews.

Main Results:

• Thirty-nine studies were included, yielding 285 distinct outcomes categorized into 29 domains.
• Common outcome areas included clinical response, safety, and resource use. Investigator-reported outcomes predominated (85%), with limited patient-reported outcomes (8%).
• A variety of OMIs were used, with significant diversity noted due to MMP's heterogeneous presentation.

Conclusions:

• Standardized, consensus-based outcome definitions and high-quality instruments are essential for improving MMP research efficacy.
• Incorporating patient perspectives and critically appraising OMI measurement properties are key recommendations for future studies.