Harvard Celiac Disease Research Program, Department of Medicine, Division of Gastroenterology, Beth Israel Deaconess Medical Center, 330 Brookline Avenue, Boston, MA 02215, USA.
Harvard Celiac Disease Research Program, Beth Israel Deaconess Medical Center, 330 Brookline Avenue, Boston, MA 02215, USA; Division of Gastroenterology and Nutrition, Boston Children's Hospital, 300 Longwood Avenue, Boston, MA 02215, USA.
Gastroenterol Clin North Am. 2019 Mar;48(1):85-99. doi: 10.1016/j.gtc.2018.09.006. Epub 2018 Dec 13.
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.
目前，针对乳糜泻的诊断和治疗干预措施仍存在未满足的需求。临床试验和真实世界研究都需要仔细选择临床结局指标。通常情况下，血清学或组织学均不是合适的主要结局指标。本文综述了各种肠功能和营养指标、症状和健康相关生活质量的患者报告结局指标，以及用于乳糜泻干预研究的疾病负担衡量指标。通过一系列案例研究，说明了在选择饮食干预、药物干预和真实世界研究的结局指标时需要考虑的关键问题。
