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

2022

2022 Mar 02

1097-6833

<p><strong>OBJECTIVES: </strong>To identify pediatric patient-reported outcomes (PROs) that are associated with chronic conditions and to evaluate the effects of chronic disease activity on PROs.</p>

<p><strong>STUDY DESIGN: </strong>Participants 8-24 years-old and their parents were enrolled into 14 studies that evaluated PROMIS® 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 United States general population of children using nationally representative percentiles. PRO-specific coefficients of variation were computed to illustrate the degree of variation in scores within versus between conditions. Condition-specific measures of disease severity and Cohens d effect sizes were used to examine PRO scores by disease activity.</p>

<p><strong>RESULTS: </strong>Participants included 2,975 child respondents and 2,392 parent respondents who provided data for 3,409 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.</p>

<p><strong>CONCLUSIONS: </strong>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.</p>

10.1016/j.jpeds.2022.02.052

J Pediatr

35247394

The Childhood Arthritis & Rheumatology Research Alliance Start Time Optimization of Biologics in Polyarticular Juvenile Idiopathic Arthritis Study

2021

2021 Jun 08

2326-5205

<p><strong>BACKGROUND: </strong>The optimal time to start biologics for polyarticular JIA (pJIA) remains uncertain. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed 3 consensus treatment plans (CTPs) for untreated pJIA to compare strategies for starting biologics.</p>

<p><strong>METHODS: </strong>Start Time Optimization of biologics in PJIA (STOP-JIA) was a prospective, observational, CARRA Registry study comparing the effectiveness of: 1) Step Up (SU)- initial non-biologic disease modifying anti-rheumatic drug (DMARD) monotherapy, adding biologic if needed; 2) Early Combination (EC)- DMARD and biologic started together; 3) Biologic First (BF)- biologic monotherapy. The primary outcome was clinically inactive disease (CID) (Wallace) off glucocorticoids at 12 months. Secondary outcomes included PROMIS® pain interference and mobility, inactive disease defined by the clinical Juvenile Arthritis Disease Activity Score (cJADAS10 ID) and pediatric ACR70 (pACR70).</p>

<p><strong>RESULTS: </strong>Of 400 patients enrolled, 257 (64%) began SU, 100 (25%) EC and 43 (11%) BF. After propensity score weighting and multiple imputation, 37% of EC, 32% SU and 24% BF achieved ACR CID (p=0.17). cJADAS10 ID (score ≤2.5) also favored EC over SU (59% versus 43%; p=0.03) as did pACR70 results (80% versus 64%; p=0.008) but generalizability is limited by missing data. PROMIS® measures improved in all groups, but without significant differences. Seventeen serious adverse events were reported (mostly infections).</p>

<p><strong>CONCLUSIONS: </strong>Achievement of CID off GC did not significantly differ between groups at 12 months. While there was a statistically significant higher likelihood of EC achieving cJADAS10 ID and pACR70, these results require further exploration.</p>

10.1002/art.41888

Arthritis Rheumatol

34105312

Juvenile Spondyloarthritis in the CARRA Registry: High Biologic Use, Low Prevalence of HLA-B27, and Equal Sex Representation in Sacroiliitis.

2020

2020 Dec 16

2151-4658

<p><strong>OBJECTIVE: </strong>To describe characteristics of children with enthesitis-related arthritis (ERA) and juvenile psoriatic arthritis (JPsA) enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry.</p>

<p><strong>METHODS: </strong>All children with ERA and JPsA were identified. Demographics, clinical characteristics, and treatments were described. Those with and without sacroiliitis were compared. In those with sacroiliitis, the first visit with clinically active sacroiliitis (which came first in 72% of cases) was compared to the first visit without.</p>

<p><strong>RESULTS: </strong>Nine hundred two children with ERA or JPsA were identified. Children with ERA were older at diagnosis (10.8 vs. 8.2 years, p&lt;0.01) and more likely male (56% vs. 38%, p&lt;0.01). Polyarticular involvement was reported in 57% and 72% of those with ERA and JPsA. HLA-B27 was positive in 38% and 12% of those tested with ERA and JPsA. At least one biologic was taken by 72% and 64% of those with ERA and JPsA. Sacroiliitis (diagnosed clinically and/or by imaging) was reported in 28% (40% ERA and 12% JPsA). Of these, 54% were female, 36% were HLA-B27 positive, and 81% took at least one biologic. In children with sacroiliitis, the physician global, parent/patient global, and cJADAS 10 were all significantly worse at the first visit with clinically active sacroiliitis versus the first visit without active sacroiliitis.</p>

<p><strong>CONCLUSION: </strong>In this registry, there are over 900 children with ERA or JPsA. There was high biologic use in this population, especially in those with sacroiliitis. Further, there was equal sex representation in those with sacroiliitis.</p>

10.1002/acr.24537

Arthritis Care Res (Hoboken)

33331139

The Childhood Arthritis & Rheumatology Research Alliance Consensus Treatment Plans: Towards Comparative Effectiveness in the Pediatric Rheumatic Diseases.

2018

2018 Jan 15

2326-5205

<p>The pediatric rheumatic diseases are a heterogeneous group of rare diseases, posing a number of challenges for the use of traditional clinical and translational research approaches. Innovative comparative effectiveness approaches are needed to efficiently study treatment approaches and disease outcomes. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed the consensus treatment plan (CTP) approach as a comparative effectiveness tool for research in pediatric rheumatology. CTPs are treatment strategies, developed by consensus methods among CARRA members, intended to reduce variation in treatment approaches, standardize outcome measurements, and allow for comparison of the effectiveness of different approaches with the goal of improving disease outcomes. To date, CTPs have been published for 7 different diseases and disease manifestations. The approach has been successfully piloted for juvenile localized scleroderma, systemic onset juvenile idiopathic arthritis (JIA), polyarticular JIA, dermatomyositis, and lupus nephritis. Large-scale studies are underway for systemic JIA and polyarticular JIA, with the CARRA patient registry serving as the data collection platform. These studies have been designed with stakeholder involvement, including active input from CARRA providers, patients, and parents, with the goal of increasing the feasibility and ensuring the relevance of the outcomes. These studies include ancillary biospecimen collection intended to support additional translational and mechanistic studies. Data from these ongoing CTP studies will provide more information on the ability of this approach to identify effective treatment strategies and improve outcomes for the pediatric rheumatic diseases. This article is protected by copyright. All rights reserved.</p>

10.1002/art.40395

29333701

Pilot study comparing the Childhood Arthritis & Rheumatology Research Alliance (CARRA) systemic Juvenile Idiopathic Arthritis Consensus Treatment Plans.

2017

23

2017 Apr 11

1546-0096

<p><strong>OBJECTIVES: </strong>To assess the feasibility of studying the comparative effectiveness of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) consensus treatment plans (CTPs) for systemic Juvenile Idiopathic Arthritis (JIA) using an observational registry.</p>

<p><strong>METHODS: </strong>Untreated systemic JIA patients enrolled in the CARRA Registry were begun on one of 4 CTPs chosen by the treating physician and patient/family (glucocorticoid [GC] alone; methotrexate [MTX] ± GC; IL1 inhibitor [IL1i] ± GC; IL6 inhibitor [IL6i] ± GC). The primary outcome of clinical inactive disease (CID) without current GC use was assessed at 9&nbsp;months.</p>

<p><strong>TRIAL REGISTRATION: </strong>clinicaltrials.gov NCT01697254; first registered 9/28/12 (retrospectively enrolled).</p>

<p><strong>RESULTS: </strong>Thirty patients were enrolled at 13 sites; eight patients were started on a non-biologic CTP (2 GC, 6 MTX) and 22 patients on a biologic CTP (12 IL1i, 10 IL6i) at disease onset. Demographic and disease features were similar between CTP groups. CTP choice appeared to segregate by site preference. CID off GC was achieved by 37% (11 of 30) including 11/22 (50%) starting a biologic CTP compared to 0/8 starting a non-biologic CTP (p = 0.014). There were four serious adverse events: two infections, one appendicitis and one macrophage activation syndrome.</p>

<p><strong>CONCLUSIONS: </strong>The CARRA systemic JIA CTP pilot study demonstrated successful implementation of CTPs using the CARRA registry infrastructure. Having demonstrated feasibility, a larger study using CTP response to better determine the relative effectiveness of treatments for new-onset systemic JIA is now underway.</p>

10.1186/s12969-017-0157-1

Pediatr Rheumatol Online J

28399931

Identifying Targets for Improving Mental Healthcare of Adolescents with Systemic Lupus Erythematosus: Perspectives from Pediatric Rheumatology Clinicians in the United States and Canada.

2016

2016 Apr 1

0315-162X

<p><strong>OBJECTIVE: </strong>To identify targets for improving mental healthcare of adolescents with systemic lupus erythematosus (SLE) by assessing current practices and perceived barriers for mental health intervention by pediatric rheumatology clinicians.</p>

<p><strong>METHODS: </strong>Members of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) completed a Web-based survey assessing current mental health practices, beliefs, and barriers. We examined associations between provider characteristics and the frequency of barriers to mental health screening and treatment using multivariable linear regression.</p>

<p><strong>RESULTS: </strong>Of the 375 eligible CARRA members, 130 responded (35%) and 119 completed the survey. Fifty-two percent described identification of depression/anxiety in adolescents with SLE at their practice as inadequate, and 45% described treatment as inadequate. Seventy-seven percent stated that routine screening for depression/anxiety in pediatric rheumatology should be conducted, but only 2% routinely used a standardized instrument. Limited staff resources and time were the most frequent barriers to screening. Respondents with formal postgraduate mental health training, experience treating young adults, and practicing at sites with very accessible mental health staff, in urban locations, and in Canada reported fewer barriers to screening. Long waitlists and limited availability of mental health providers were the most frequent barriers to treatment. Male clinicians and those practicing in the Midwest and Canada reported fewer barriers to treatment.</p>

<p><strong>CONCLUSION: </strong>Pediatric rheumatology clinicians perceive a need for improved mental healthcare of adolescents with SLE. Potential strategies to overcome barriers include enhanced mental health training for pediatric rheumatologists, standardized rheumatology-based mental health practices, and better integration of medical and mental health services.</p>

10.3899/jrheum.151228

J. Rheumatol.

27036378

Enthesitis-related arthritis is associated with higher pain intensity and poorer health status in comparison with other categories of juvenile idiopathic arthritis: the Childhood Arthritis and Rheumatology Research Alliance Registry.

2012

2341-51

2012 Dec

0315-162X

<p><strong>OBJECTIVE: </strong>To assess the relative effect of clinical factors and medications on pain intensity, physical function, and health status in juvenile idiopathic arthritis (JIA).</p>

<p><strong>METHODS: </strong>We conducted a retrospective cross-sectional study of data from children with JIA enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry. We tested whether clinical characteristics of JIA were associated with pain intensity, physical function, and health status using multivariable linear and ordinal logistic regression.</p>

<p><strong>RESULTS: </strong>During the study period, 2571 subjects with JIA enrolled in the CARRA Registry. Ratings of pain intensity, physical function, and health status differed significantly between JIA categories. In comparison to other categories of JIA, subjects with enthesitis-related arthritis (ERA) reported worse pain and function. In multivariable analyses, higher active joint count and current use of nonsteroidal antiinflammatory drugs (NSAID), biologics, or corticosteroids were associated with worse scores on all patient-reported measures. ERA and older age were significantly associated with higher pain intensity and poorer health status. Systemic JIA and uveitis were significantly associated with worse health status. Enthesitis, sacroiliac tenderness, and NSAID use were independently associated with increased pain intensity in ERA. The correlation was low between physician global assessment of disease activity and patient-reported pain intensity, physical function, and health status.</p>

<p><strong>CONCLUSION: </strong>Significant differences in pain intensity, physical function, and health status exist among JIA categories. These results suggest that current treatments may not be equally effective for particular disease characteristics more common in specific JIA categories, such as enthesitis or sacroiliac tenderness in ERA.</p>

10.3899/jrheum.120642

J. Rheumatol.

23070991