(Outcome) Measure for (Intervention) Measures: A Guide to Choosing the Appropriate Noninvasive Clinical Outcome Measure for Intervention Studies in Celiac Disease

Abstract

There is an unmet need for diagnostic and treatment interventions for celiac disease. Both clinical trials and real-world studies require careful selection of clinical outcome measures. Often, neither serology nor histology is an appropriate primary outcome. This article reviews various measures of intestinal function and nutrition, patient-reported outcome measures for symptoms and for health-related quality of life, and measures of sickness burden as they apply to intervention studies for celiac disease. A series of case studies is presented to illustrate key considerations in selecting outcome measures for dietary interventions, pharmacologic interventions, and real-world studies.

Keywords: Celiac disease; Intervention studies; Outcome measures; Patient-reported outcomes; Study design.