Varni James W, Franciosi James P, Shulman Robert J, Saeed Shehzad, Nurko Samuel, Neigut Deborah A, Bendo Cristiane B, Patel Ashish S, Self Mariella M, Saps Miguel, Zacur George M, Denham Jolanda, Dark Chelsea Vaughan, Pohl John F
1Department of Pediatrics, College of Medicine, Texas A&M University, College Station, Texas; 2Department of Landscape Architecture and Urban Planning, College of Architecture, Texas A&M University, College Station, Texas; 3Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; 4Department of Pediatrics, Baylor College of Medicine, Children's Nutrition Research Center, Texas Children's Hospital, Houston, Texas; 5Center for Motility and Functional Gastrointestinal Disorders, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts; 6Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital Colorado, Aurora, Colorado; 7Department of Pediatric Dentistry and Orthodontics, Faculty of Dentistry, Federal University of Minas Gerais, Belo Horizonte, Brazil; 8Division of Pediatric Gastroenterology, Children's Medical Center of Dallas, University of Texas Southwestern Medical School, Dallas, Texas; 9Departments of Psychiatry and Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, Texas; 10Division of Gastroenterology, Hepatology and Nutrition, Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois; 11Division of Pediatric Gastroenterology, Nationwide Children's Hospital, Ohio State University School of Medicine, Columbus, Ohio; 12Department of Psychology, Texas A&M University, College Station, Texas; and 13Department of Pediatric Gastroenterology, Primary Children's Hospital, University of Utah, Salt Lake City, Utah (Drs. Franciosi and Denham are now with the Division of Gastroenterology, Hepatology and Nutrition, Nemours Children's Hospital, Orlando, Florida. Dr. Saps is now with the Division of Pediatric Gastroenterology, Nationwide Children's Hospital, Ohio State University, Columbus, Ohio. Dr. Zacur is now with the Division of Pediatric Gastroenterology, C.S. Mott Children's Hospital, University o
Inflamm Bowel Dis. 2015 May;21(5):1115-24. doi: 10.1097/MIB.0000000000000351.
Patient-reported outcomes are essential in determining the broad impact of inflammatory bowel disease (IBD) and treatments from the patient's perspective. The primary study objectives were to compare the gastrointestinal symptoms and worry of pediatric patients with IBD with matched healthy controls and to compare Crohn's disease and ulcerative colitis with each other using the Pediatric Quality of Life Inventory (PedsQL) Gastrointestinal Symptoms and Gastrointestinal Worry Scales.
PedsQL Gastrointestinal Symptoms and Worry Scales were completed in a 9-site study by 256 pediatric patients with IBD and 259 parents of patients (263 families; Crohn's disease [n = 195], ulcerative colitis [n = 68]). Ten Gastrointestinal Symptoms Scales measuring stomach pain, stomach discomfort when eating, food and drink limits, trouble swallowing, heartburn and reflux, nausea and vomiting, gas and bloating, constipation, blood in poop, and diarrhea were administered along with 2 Gastrointestinal Worry Scales. A matched group of 384 healthy children families completed the PedsQL in an Internet survey.
PedsQL Gastrointestinal Symptoms and Worry Scales distinguished between pediatric patients with IBD in comparison with healthy controls (P < 0.001), with larger effect sizes for symptoms indicative of IBD, supporting known-groups validity and clinical interpretability including minimal important difference scores. Patients with Crohn's disease or ulcerative colitis did not demonstrate significantly different gastrointestinal symptoms or worry in comparison with each other.
The PedsQL Gastrointestinal Symptoms and Worry Scales may be used as common metrics across pediatric patients with IBD, including Crohn's disease and ulcerative colitis separately to measure gastrointestinal-specific symptoms in clinical research and practice.
患者报告的结局对于从患者角度确定炎症性肠病（IBD）及其治疗的广泛影响至关重要。主要研究目标是使用儿童生活质量量表（PedsQL）胃肠道症状和胃肠道担忧量表，比较IBD儿科患者与匹配的健康对照的胃肠道症状和担忧，并比较克罗恩病和溃疡性结肠炎。
在一项9个地点的研究中，256名IBD儿科患者和259名患者家长（263个家庭；克罗恩病[n = 195]，溃疡性结肠炎[n = 68]）完成了PedsQL胃肠道症状和担忧量表。同时使用10个胃肠道症状量表测量胃痛、进食时胃部不适、饮食限制、吞咽困难、烧心和反流、恶心和呕吐、气体和腹胀、便秘、便血和腹泻，以及2个胃肠道担忧量表。一组匹配的384名健康儿童家庭在互联网调查中完成了PedsQL。
与健康对照相比，PedsQL胃肠道症状和担忧量表能够区分IBD儿科患者（P < 0.001），对于指示IBD的症状具有更大的效应量，支持已知组效度和临床可解释性，包括最小重要差异分数。与彼此相比，克罗恩病患者或溃疡性结肠炎患者未表现出明显不同的胃肠道症状或担忧。
PedsQL胃肠道症状和担忧量表可作为IBD儿科患者的通用指标，包括分别用于克罗恩病和溃疡性结肠炎患者，以在临床研究和实践中测量胃肠道特异性症状。
