Clinical Presentation and Five-Year Therapeutic Management of Very Early-Onset Inflammatory Bowel Disease in a Large North American Cohort.

OBJECTIVE: To evaluate the presentation, therapeutic management, and long-term outcome of children with very early-onset (VEO) (≤ 5 years of age) inflammatory bowel disease (IBD). STUDY DESIGN: Data were obtained from an inception cohort of 1928 children with IBD enrolled in a prospective observational registry at multiple centers in North America. RESULTS: One hundred twelve children were ≤ 5 years of age with no child enrolled at <1 year of age. Of those, 42.9% had Crohn's disease (CD), 46.4% ulcerative colitis (UC), and 10.7% had IBD-unclassified. Among the children with CD, children 1-5 years of age had more isolated colonic disease (39.6%) compared with 6- to 10-year-olds (25.3%, P = .04), and 11- to 16-year-olds (22.3%, P < .01). The change from a presenting colon-only phenotype to ileocolonic began at 6-10 years. Children 1-5 years of age with CD had milder disease activity (45.8%) at diagnosis compared with the oldest group (28%, P = .01). Five years postdiagnosis, there was no difference in disease activity among the 3 groups. However, compared with the oldest group, a greater proportion of 1- to 5-year-olds with CD were receiving corticosteroids (P < .01) and methotrexate (P < .01), and a greater proportion of 1- to 5-year-olds with UC were receiving mesalamine (P < .0001) and thiopurine immunomodulators (P < .0002). CONCLUSIONS: Children with VEO-CD are more likely to have mild disease at diagnosis and present with a colonic phenotype with change to an ileocolonic phenotype noted at 6-10 years of age. Five years after diagnosis, children with VEO-CD and VEO-UC are more likely to have been administered corticosteroids and immunomodulators despite similar disease activity in all age groups. This may suggest development of a more aggressive disease phenotype over time.

Development and validation of the pediatric liver transplantation quality of life: a disease-specific quality of life measure for pediatric liver transplant recipients.

OBJECTIVES: To develop and validate a Pediatric Liver Transplantation Quality of Life (PeLTQL) questionnaire via an international multicenter collaboration. STUDY DESIGN: Item generation with 146 child and/or parent interviews (92 pediatric liver transplantation [LT] recipients) and 3 focus groups generated over 300 items. An item reduction questionnaire with 76 questions was completed by 320 participants (212 pediatric LT recipients). RESULTS: Frequency-importance product ranking, questionnaire formatting, and pre-testing resulted in a 26-item PeLTQL questionnaire. Factor analysis identified 3 domains: future health, coping and adjustment, and social-emotional. The validation phase was completed by 133 (46% male) LT recipients (aged 8-18 years). Internal consistency (Cronbach α = 0.86) and test-retest reliability (intraclass correlation coefficient = 0.85) were excellent. Mean patient PeLTQL score was 69.54 ± 13.06. Construct validity with validated tools identified significant correlations between mean PeLTQL scores and (1) Pediatric Quality of Life Inventory generic (r = 0.64, P < .001); (2) Pediatric Quality of Life Inventory transplant (r = 0.73, P < .001); and (3) Screen for Child Anxiety Related Disorders (r = -0.57, P < .001) scores. Only 17/3458 (0.5%) questions were left blank. A Flesch-Kincaid grade level of 5.4 was calculated as a measure of the PeLTQL readability statistic. CONCLUSIONS: The PeLTQL is a valid and reliable novel 26-item disease-specific health related quality of life instrument for LT recipients aged 8-18 years. Low PeLTQL scores can identify patients at risk for childhood anxiety and depression. The tool is now ready for broad use in both clinical practice and clinical interventional trials.

Do parents and children agree? Quality-of-life assessment of children with inflammatory bowel disease and their parents.

BACKGROUND AND OBJECTIVES: Multi-item measures of inflammatory bowel disease (IBD) activity based on clinical, laboratory, and/or endoscopic variables do not take into consideration the impact on the patients' emotional aspects and adaptation to the disease. The aim of the present study was to evaluate concordance between parent and child ratings of health-related quality of life on the IMPACT-III questionnaire in children with IBD. METHODS: The IMPACT-III questionnaire was used to measure quality of life in 27 patients (mean age 14.2 ± 3 years, 40% girls) and one of their parents (82% mothers). Most of the patients had inactive disease at the time of the study. Differences between parent-proxy ratings and child ratings on the IMPACT-III were compared via paired-samples t tests, intraclass correlation coefficients, and standardized difference scores. RESULTS: Parent-proxy and patient ratings were similar on total IMPACT-III and its related domains (bowel symptoms, systemic symptoms, social functioning, body image, treatment/interventions), except that significant differences on emotional functioning ratings were found (P = 0.003). Intraclass correlation coefficients showed medium-to-large effect sizes (range 0.52-0.88) and standardized difference scores showed varying degrees of bias depending on the domain measured (range -0.64 to 0.32). CONCLUSIONS: Parents served as a good proxy for quality-of-life ratings in this population of pediatric patients with IBD. The degree of concordance between parent and child scores, however, varied, as observed in the present study in which parents underreported their child's health-related quality of life on the IMPACT-III emotional functioning domain.

Body mass index in children with newly diagnosed inflammatory bowel disease: observations from two multicenter North American inception cohorts.

OBJECTIVE: To conduct a systematic review of children with newly diagnosed inflammatory bowel disease (IBD) from 2 prospective inception cohorts to examine body mass index (BMI) status at presentation. STUDY DESIGN: Clinical, demographic, and BMI data were obtained from 783 patients with newly diagnosed IBD. National Health and Nutrition Examination Survey data for 2748 healthy children were used as a control. RESULTS: Most children with Crohn's disease and ulcerative colitis had a BMI in the normative range (5%-84%). Low BMI (<5%) was seen in 22% to 24% of children with Crohn's disease and 7% to 9% of children with ulcerative colitis. Ten percent of children with Crohn's disease and 20% to 30% of children with ulcerative colitis had a BMI at diagnosis consistent with overweight or risk for overweight. CONCLUSION: Children with IBD are affected by current population trends toward overweight. A significant subgroup of children with newly diagnosed IBD has a BMI categorized as overweight or at risk for overweight. Clinicians should be aware of possible IBD diagnosis in the presence increased BMI.