Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases.

OBJECTIVES: To identify pediatric patient-reported outcomes (PROs) that are associated with chronic conditions and to evaluate the effects of chronic disease activity on PROs. STUDY DESIGN: Participants (8-24 years old) and their parents were enrolled into 14 studies that evaluated Patient-Reported Outcome Measurement Information System PROs across 10 chronic conditions-asthma, atopic dermatitis, cancer, cancer survivors, chronic kidney disease, Crohn's disease, juvenile idiopathic arthritis, lupus, sickle cell disease, and type 1 diabetes mellitus. PRO scores were contrasted with the US general population of children using nationally representative percentiles. PRO-specific coefficients of variation were computed to illustrate the degree of variation in scores within vs between conditions. Condition-specific measures of disease severity and Cohen d effect sizes were used to examine PRO scores by disease activity. RESULTS: Participants included 2975 child respondents and 2392 parent respondents who provided data for 3409 unique children: 52% were 5-12 years old, 52% female, 25% African American/Black, and 14% Hispanic. Across all 10 chronic conditions, children reported more anxiety, fatigue, pain, and mobility restrictions than the general pediatric population. Variation in PRO scores within chronic disease cohorts was equivalent to variation within the general population, exceeding between-cohort variation by factors of 1.9 (mobility) to 5.7 (anxiety). Disease activity was consistently associated with poorer self-reported health, and these effects were weakest for peer relationships. CONCLUSIONS: Chronic conditions are associated with symptoms and functional status in children and adolescents across 10 different disorders. These findings highlight the need to complement conventional clinical evaluations with those obtained directly from patients themselves using PROs.

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

OBJECTIVES: To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness. STUDY DESIGN: We evaluated the responsiveness of PROMIS pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical trial in pediatric patients with Crohn's disease (CD). We examined the relationships between changes in PROMIS pediatric measures and changes in disease activity by evaluating PRO score changes among patients who did and patients who did not experience improvement in disease activity. RESULTS: Participants included 266 children and adolescents with CD from a total of 35 institutions. Over the course of follow-up, participants showed improvement in most PRO domains, with the largest effect sizes observed for the clinically improved group. Patients who maintained steroid-free remission showed significantly lower PRO scores for the Pain Interference, Fatigue, and inflammatory bowel disease (IBD) Symptoms domains and higher scores for the Positive Affect domain. CONCLUSIONS: This study demonstrates the responsiveness of the PROMIS pediatric measures of Fatigue and Pain Interference as study endpoints in a large, multicenter pragmatic trial in pediatric CD, extending a growing body of research supporting the use of PROMIS pediatric measures as reliable PRO endpoints for clinical trials.

Association of Care Coordination Experience and Health Services Use with Main Provider Type for Children with Inflammatory Bowel Disease.

OBJECTIVES: To describe care coordination experience for families of children with inflammatory bowel disease (IBD) and compare use of health services between families who identified a primary care physician (PCP) vs a gastroenterologist as a child's main provider. STUDY DESIGN: This is a cross-sectional survey of care coordination experiences and health services use for children 6-19 years old receiving care in the IBD program at a children's hospital during 2018. English-speaking parents completed the Family Experiences with Coordination of Care Survey about their child's main provider and reported past-year health services. Bivariate testing and multivariate logistic regression explored differences in care coordination experience and health services by main provider, adjusted for demographic and clinical variables. RESULTS: A total of 113 of 270 (42%) invited patients participated. Among 101 patients with complete data, 41% identified a PCP main provider. Performance on 5 of 16 Family Experiences with Coordination of Care indicators was higher for patients reporting a gastroenterologist vs a PCP main provider. However, having a PCP vs gastroenterologist main provider was associated with greater use of any past-year primary care services (adjusted proportion 94% vs 75%; P = .01) and of mental health services when needed (95% vs 60%; P < .01). Need for IBD-related hospitalization and emergency department visits did not differ between groups. CONCLUSIONS: Children with IBD may experience trade-offs in care coordination quality and important, non-disease-focused health services based on whom parents perceive as the main provider. Efforts to enhance cross-team coordination among families and primary and specialty care teams are needed to improve overall care quality.

Body Image Dissatisfaction among Pediatric Patients with Inflammatory Bowel Disease.

OBJECTIVES: To determine risk factors for body image dissatisfaction among pediatric patients with inflammatory bowel disease (IBD). STUDY DESIGN: We performed a cross-sectional study of children aged 9-18 years in the IBD Partners Kids & Teens cohort. Participants completed surveys including demographics, disease characteristics and activity indices, and psychosocial outcomes measured by IMPACT-III questionnaires. We defined body image dissatisfaction if participants answered "I look awful" or "I look bad." Bivariate analyses assessed associations between body image dissatisfaction and demographic, disease-related and psychosocial factors; logistic regression models evaluated associations between risk factors and body image dissatisfaction. RESULTS: IMPACT-III was completed by 664 patients, with 74 (11.1%) reporting body image dissatisfaction. Patients with body image dissatisfaction were more likely to be female (P < .01), older (median age 15 vs 13 years, P < .01), and diagnosed with IBD at an older age (12 vs 10 years, P < .01). Those with body image dissatisfaction had greater body mass index percentile (P = .02), more active disease (P < .01), more current steroid use (P < .01), and more depression and anxiety (P < .01). Female sex (OR 2.31; 95% CI 1.22-4.39), depression (OR 4.73; 95% CI 2.41-9.26), and anxiety (OR 5.42; 95% CI 2.48-11.80) were independently associated with body image dissatisfaction. CONCLUSIONS: In this cohort, risk factors for body image dissatisfaction include female sex, older age at diagnosis, active disease, current steroid use, greater body mass index, and comorbid mood disorder. Interventions targeting modifiable risk factors for body image dissatisfaction may improve quality of life in pediatric patients with body image dissatisfaction.

Black/African American Patients with Pediatric Crohn's Disease Report Less Anxiety and Fatigue than White Patients.

OBJECTIVES: To compare patient-reported outcomes in black/African American patients with white patients participating in IBD Partners Kids & Teens, in order to identify possible racial healthcare disparities in pediatric inflammatory bowel disease (IBD) as future targets for improvement. STUDY DESIGN: This was a cross-sectional analysis comparing patient-reported outcomes in black/African American patients with white patients, aged 9-18 years, with IBD participating in the IBD Partners Kids & Teens cohort from August 2013 to April 2018. Secondary outcomes included number of IBD-related hospitalizations and surgeries, current medication use, and disease activity. RESULTS: We included 401 patients with Crohn's disease (white = 378 [94%]; black/African American = 23 [6%]). For children with Crohn's disease, black/African American patients compared with white patients reported less anxiety (40.7 vs 47.5, P = .001) and fatigue (44.3 vs 48.4, P = .047) despite more frequently reported treatment with biologics (91% vs 61%, P = .006) and antibiotics (17% vs 5%, P = .03) and history of hospitalizations (81% vs 52%, P = .02). CONCLUSIONS: Black/African American children with Crohn's disease were less likely to report anxiety or fatigue than white patients, despite an apparent more severe disease course reflected by greater reported frequency of treatment with biologics and antibiotics and history of hospitalizations.

The Burden of Hospital Readmissions among Pediatric Patients with Inflammatory Bowel Disease.

OBJECTIVE: To evaluate the burden and predictors of hospital readmissions among pediatric patients with inflammatory bowel disease using the Nationwide Readmissions Database. STUDY DESIGN: We performed a retrospective cohort study using 2013 Nationwide Readmissions Database. International Classification of Diseases, Ninth Revision, Clinical Modification codes were used to identify patients <18 years with diagnoses of ulcerative colitis (UC) or Crohn's disease (CD). Demographic factors and details of hospitalizations were evaluated using survey procedures in SAS v 9.4 (SAS Institute, Cary, North Carolina). Multivariable logistic regression was used to estimate ORs and 95% CIs of readmission. RESULTS: Among 2733 hospitalizations (63% CD, 37% UC), 611 (22%) patients were readmitted within 90 days of the index hospitalization. Readmission resulted in weighted estimates of 11 440 excess days of hospitalization and total charges of over $107 million. For CD, male sex (aOR 1.36, 95% CI 1.03-1.81) and co-existing anxiety or depression (aOR 1.89, 95% CI 1.06-3.40) were associated with increased readmissions, while patients who underwent surgery had decreased readmissions (aOR 0.40, 95% CI 0.24-0.65). In patients with UC, an index admission of >7 days was associated with increased readmissions (aOR 1.69, 95% CI 1.09-2.62). CONCLUSIONS: Readmission occurs frequently in children with inflammatory bowel disease and is associated with significant cost and resource burdens. Among patients with CD, psychiatric comorbidities such as anxiety and depression are apparent drivers of readmission.

Patient-Reported Outcomes Measurement Information System in Children with Crohn's Disease.

OBJECTIVES: To assess the criterion validity and responsiveness of Patient-Reported Outcomes Measurement Information System (PROMIS) in a web-based cohort of children with Crohn's disease. STUDY DESIGN: We recruited children with Crohn's disease (ages 9-17 years) and their parents from the web-based Crohn's and Colitis Foundation of America Kids and Teens Study cohort. Upon entry into the cohort and 6 months later, children self-reported Crohn's disease activity, health-related quality of life, and PROMIS domains of pain interference, anxiety, depression, fatigue, and peer relationships. RESULTS: Mean PROMIS scores for the 276 participating patients were worse among those with worse self-reported Crohn's disease activity (per Short Crohn's Disease Activity Index, P < .005 for all), Crohn's disease activity in the prior 6 months (per Manitoba Index, P < .01 for all), and health-related quality of life (per IMPACT-35, P < .001 for all). One hundred forty-three patients and their parents completed follow-up questionnaires, 75% of whom reported stable disease activity. Those with improved Crohn's disease activity reported improved PROMIS scores, and those with worsened Crohn's disease activity reported worse PROMIS scores for all domains except anxiety. All participants reported improved anxiety from baseline, but those with stable or worsened Crohn's disease activity reported less improvement (P = .07). CONCLUSIONS: PROMIS scores were significantly associated with Crohn's disease activity in a linear and clinically meaningful manner, and responded to change in Crohn's disease activity over a 6-month period. This supports the criterion validity and responsiveness of pediatric PROMIS.

Healthcare utilization and comorbidity burden among children and young adults in the United States with systemic lupus erythematosus or inflammatory bowel disease.

OBJECTIVE: We sought to assess the feasibility of using a health insurance claims database to estimate the prevalence and health care utilization and costs among children diagnosed with systemic lupus erythematosus (SLE) and inflammatory bowel disease (IBD). STUDY DESIGN: This was a retrospective analysis of the LifeLink insurance claims database for the years 2000-2006. Children (0-15 years) and young adults (16-25 years) with ≥ 2 diagnosis claims for SLE or IBD were selected as the 2 cohorts of interest. For each member of the SLE and IBD cohorts, 2 individuals were randomly selected for a matched comparison group. All the analyses were descriptive in nature, CI for differences between means and 2 proportions for measures including health care utilization, comorbidity burden were based on t tests and 2-group tests of proportions. RESULTS: We identified 278 patients with SLE (prevalence estimate: 7.9 per/100000 population) and 1174 patients with IBD (33.2 per/100000 population). The mean annual total medical costs was substantially higher for the SLE (difference: $22223; 95% CI: $14961-$29485) and IBD (difference: $16238; 95% CI: $14395-$18082) cohorts compared with those of the comparator cohort. We observed higher comorbidity burdens in the SLE and IBD cohorts than we saw in the comparator cohort. CONCLUSIONS: Administrative claims data can be a useful tool for assessing the comparative prevalence and associated resource utilization of rare conditions such as SLE and IBD.