Development and validation of a celiac disease quality of life instrument for North American children.

OBJECTIVE: Given the social constraints imposed by a gluten-free diet, it can be hypothesized that children with celiac disease (CD) living in the United States have a reduced health-related quality of life (HRQOL); however, there is no validated CD-specific HRQOL instrument for children living in the United States. The goals of this study were to develop and validate a CD-specific HRQOL instrument for children 8 to 18 years of age with CD and to report HRQOL in these children using both generic- and disease-specific instruments. METHODS: This was a prospective study using focus group methodology to develop a CD-specific HRQOL instrument that was then administered to children 8 to 18 years of age with CD living throughout the United States. Instrument validation methods included construct, convergent, and divergent validities. RESULTS: Two instruments were developed: CD-specific pediatric HRQOL instrument (CDPQOL) 8 to 12 and CDPQOL 13 to 18. A total of 181 children with CD completed the CDPQOL as well as a comparator generic instrument. Exploratory factor analysis restructured the CDPQOL and reduced the total number of items. The CDPQOL showed a moderate agreement with the Psychosocial dimensions of the generic instrument confirming convergent validity and low-to-moderate agreement with the Physical Health Summary dimension of the generic instrument confirming divergent validity. CONCLUSIONS: The CDPQOL, consisting of 13 to 17 questions, is a validated instrument for the measurement of HRQOL in children 8 to 18 years of age with CD living in the United States.

Celiac disease in normal-weight and overweight children: clinical features and growth outcomes following a gluten-free diet.

OBJECTIVES: There are few data on pediatric celiac disease in the United States. The aim of our study was to describe the presentation of celiac disease among children with a normal and an elevated body mass index (BMI) for age, and to study their BMI changes following a gluten-free diet (GFD). PATIENTS AND METHODS: One hundred forty-two children (age 13 months-19 years) with biopsy-proven celiac disease, contained in a registry of patients studied at our center from 2000 to 2008, had follow-up growth data available. Patients' height, weight, and BMI were converted to z scores for age and grouped by BMI as underweight, normal, and overweight. Compliance was confirmed using results of serological assays, and data of noncompliant patients were analyzed separately. Data were analyzed during the observation period and were expressed as change in height, weight, and BMI z score per month of dietary treatment. RESULTS: Nearly 19% of patients had an elevated BMI at diagnosis (12.6% overweight, 6% obese) and 74.5% presented with a normal BMI. The mean duration of follow-up was 35.6 months. Seventy-five percent of patients with an elevated BMI at diagnosis decreased their BMI z scores significantly after adherence to a GFD, normalizing it in 44% of cases. Of patients with a normal BMI at diagnosis, weight z scores increased significantly after treatment, and 13% became overweight. CONCLUSIONS: Both normal weight and overweight frequently occur in North American children presenting with celiac disease. A GFD may have a beneficial effect upon the BMI of overweight and obese children with celiac disease.

Evidence-Informed Expert Recommendations for the Management of Celiac Disease in Children.

Although the need for effective long-term follow-up for patients with celiac disease (CD) has been recognized by many expert groups, published practice guidelines have not provided a clear approach for the optimal management of these patients. In an attempt to provide a thoughtful and practical approach for managing these patients, a group of experts in pediatric CD performed a critical review of the available literature in 6 categories associated with CD to develop a set of best practices by using evidence-based data and expert opinion. The 6 categories included the following: bone health, hematologic issues, endocrine problems, liver disease, nutritional issues, and testing. Evidence was assessed by using standardized criteria for evaluating the quality of the data, grade of evidence, and strength of conclusions. Over 600 publications were reviewed, and 172 were chosen for inclusion. The thorough review of the results demonstrated that the quality of the data available was often insufficient to provide unequivocal best practices. However, using the available data and the clinical experience of the panel, a practical framework for the management of children with CD was created. These recommendations were developed by our expert panel and do not necessarily reflect the policy of the American Academy of Pediatrics. The potential usefulness of these best practices is underscored by the fact that consensus, measured by the outcome of anonymous voting, was reached by the panel for 24 of the 25 questions. We hope that these best practices may be useful to the pediatric gastroenterology and larger general pediatric communities.

Presentation of pediatric celiac disease in the United States: prominent effect of breastfeeding.

Childhood celiac disease (CD) is considered rare in the United States. Consequently there are few data concerning its clinical presentation. A validated questionnaire was distributed to families of children with CD. One hundred forty-one children with biopsy-proven CD were included in the study. We found significant differences in the clinical spectrum of children based on their infant feeding history. Exclusively breastfed children were significantly less likely to report failure to thrive (69% vs 88%, p<0.05) and short stature (37% vs 62%, p<0.05), and had a higher rate of "atypical'' symptoms (p<0.01). Breastfeeding alters the presentation and contributes to atypical presentations of CD and diagnostic delay. Pediatricians need to be aware of the diverse manifestations of celiac disease to reduce diagnostic delay.