Comparative Effectiveness of Tumor Necrosis Factor Agents and Disease-modifying Antirheumatic Therapy in Children with Enthesitis-related Arthritis: The First Year after Diagnosis.

Objective: To characterize the effect of anti-tumor necrosis factor (TNF) therapy compared to conventional synthetic disease-modifying antirheumatic drugs (csDMARD) in children with enthesitis-related arthritis (ERA) over the first year after diagnosis.

Methods: We conducted a multicenter retrospective comparative effectiveness study of children diagnosed with ERA. We estimated the effect of anti-TNF therapy on clinical variables (active joint count, tender entheses count) and patient-reported pain and global assessment of disease activity over the first year after diagnosis using state-of-the-art comparative effectiveness analytic methods.

A PROMIS Measure of Neuropathic Pain Quality.

Objectives: Neuropathic pain (NP) is a consequence of many chronic conditions. This study aimed to develop an unidimensional NP scale with scores that represent levels of NP and distinguish between individuals with NP and non-NP conditions.

Methods: A candidate item pool of 42 pain quality descriptors was administered to participants with osteoarthritis, rheumatoid arthritis, diabetic neuropathy, and cancer chemotherapy-induced peripheral neuropathy.

Validation of Patient-Reported Outcomes Measurement Information System Short Forms for Use in Childhood-Onset Systemic Lupus Erythematosus.

Objective: To validate the pediatric Patient-Reported Outcomes Measurement Information System short forms (PROMIS-SFs) in childhood-onset systemic lupus erythematosus (SLE) in a clinical setting.

Methods: At 3 study visits, childhood-onset SLE patients completed the PROMIS-SFs (anger, anxiety, depressive symptoms, fatigue, physical function-mobility, physical function-upper extremity, pain interference, and peer relationships) using the PROMIS assessment center, and health-related quality of life (HRQoL) legacy measures (Pediatric Quality of Life Inventory, Childhood Health Assessment Questionnaire, Simple Measure of Impact of Lupus Erythematosus in Youngsters [SMILEY], and visual analog scales [VAS] of pain and well-being). Physicians rated childhood-onset SLE activity on a VAS and completed the Systemic Lupus Erythematosus Disease Activity Index 2000.

Longitudinal evaluation of patient-reported outcomes measurement information systems measures in pediatric chronic pain.

The Patient-Reported Outcomes Measurement Information System (PROMIS) initiative is a comprehensive strategy by the National Institutes of Health to support the development and validation of precise instruments to assess self-reported health domains across healthy and disease-specific populations. Much progress has been made in instrument development, but there remains a gap in the validation of PROMIS measures for pediatric chronic pain. The purpose of this study was to investigate the construct validity and responsiveness to change of 7 PROMIS domains for the assessment of children (ages: 8-18) with chronic pain--Pain Interference, Fatigue, Anxiety, Depression, Mobility, Upper Extremity Function, and Peer Relationships.

Qualitative Evaluation of Pediatric Pain Behavior, Quality, and Intensity Item Candidates and the PROMIS Pain Domain Framework in Children With Chronic Pain.

Unlabelled: As initial steps in a broader effort to develop and test pediatric pain behavior and pain quality item banks for the Patient-Reported Outcomes Measurement Information System (PROMIS), we used qualitative interview and item review methods to 1) evaluate the overall conceptual scope and content validity of the PROMIS pain domain framework among children with chronic/recurrent pain conditions, and 2) develop item candidates for further psychometric testing. To elicit the experiential and conceptual scope of pain outcomes across a variety of pediatric recurrent/chronic pain conditions, we conducted 32 semi-structured individual and 2 focus-group interviews with children and adolescents (8-17 years), and 32 individual and 2 focus-group interviews with parents of children with pain. Interviews with pain experts (10) explored the operational limits of pain measurement in children.

Initial Benchmarking of the Quality of Medical Care in Childhood-Onset Systemic Lupus Erythematosus.

Objective: To assess the quality of medical care in childhood-onset systemic lupus erythematosus (SLE) at tertiary pediatric rheumatology centers as measured by observance of SLE quality indicators (SLE-QIs).

Methods: International consensus has been achieved for childhood-onset SLE-QIs capturing medical care provision in 9 domains: diagnostic testing, education of cardiovascular (CV) risk and lifestyles, lupus nephritis (LN), medication management, bone health, ophthalmologic surveillance, transition, pregnancy, and vaccination. Using medical record information, the level of performance of these childhood-onset SLE-QIs was assessed in childhood-onset SLE populations treated at 4 tertiary pediatric rheumatology centers in the US, 2 in Brazil, and 1 center in India.

PROMIS(®) pediatric self-report scales distinguish subgroups of children within and across six common pediatric chronic health conditions.

Purpose: To conduct a comparative analysis of eight pediatric self-report scales for ages 8-17 years from the National Institutes of Health (NIH) Patient Reported Outcomes Measurement Information System (PROMIS(®)) in six pediatric chronic health conditions, using indicators of disease severity.

Methods: Pediatric patients (N = 1454) with asthma, cancer, chronic kidney disease, obesity, rheumatic disease, and sickle cell disease completed items from the PROMIS pediatric mobility, upper extremity functioning, depressive symptoms, anxiety, anger, peer relationships, pain interference, and fatigue self-report scales. Comparisons within the six pediatric chronic health conditions were conducted by examining differences in groups based on the disease severity using markers of severity that were specific to characteristics of each disease.

Item-level informant discrepancies between children and their parents on the PROMIS(®) pediatric scales.

Objective: The study objective was to describe the individual item-level discrepancies between children ages 8-17 years and their parents for the PROMIS(®) pediatric scales. Contextual effects on item-level informant discrepancies for the pediatric pain interference items were further analyzed conditional on whether the child, the parent, or anyone else in the household experienced chronic pain.

Methods: Parallel pediatric self-report and parent proxy-report items were completed by approximately 300 parent-child dyads depending on form assignment and individual nonresponse.

Development and retrospective validation of the juvenile spondyloarthritis disease activity index.

Objective: To develop and validate the Juvenile Spondyloarthritis Disease Activity Index (JSpADA) for use in clinical practice and research.

Methods: Using modified Delphi consensus techniques, 10 items were selected by participants in the international pediatric rheumatology listserv, the Childhood Arthritis and Rheumatology Research Alliance, and the listserv for the pediatric section of the American College of Rheumatology. Validation was performed in a retrospective multicenter cohort of 244 children.

Childhood Arthritis and Rheumatology Research Alliance consensus treatment plans for new-onset polyarticular juvenile idiopathic arthritis.

Objective: There is no standardized approach to the initial treatment of polyarticular juvenile idiopathic arthritis (JIA) among pediatric rheumatologists. Understanding the comparative effectiveness of the diverse therapeutic options available will result in better health outcomes for polyarticular JIA. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed consensus treatment plans (CTPs) for use in clinical practice to facilitate such studies.

Item response theory, computerized adaptive testing, and PROMIS: assessment of physical function.

Objective: Patient-reported outcome (PRO) questionnaires record health information directly from research participants because observers may not accurately represent the patient perspective. Patient-reported Outcomes Measurement Information System (PROMIS) is a US National Institutes of Health cooperative group charged with bringing PRO to a new level of precision and standardization across diseases by item development and use of item response theory (IRT).

Methods: With IRT methods, improved items are calibrated on an underlying concept to form an item bank for a "domain" such as physical function (PF).

Outcomes research in childhood autoimmune diseases.

This article provides an introduction to key aspects of outcomes research in pediatric rheumatology, focusing on arthritis. Patient-centered outcomes research addresses questions of interest to multiple stakeholders in order to guide the best health care decisions suited to a particular patient's circumstances and preferences. Discussion includes the importance of maintaining high-quality longitudinal patient registries and use of valid clinical and patient-reported outcome measures.

2013 update of the 2011 American College of Rheumatology recommendations for the treatment of juvenile idiopathic arthritis: recommendations for the medical therapy of children with systemic juvenile idiopathic arthritis and tuberculosis screening among children receiving biologic medications.

Guidelines and recommendations developed and/or endorsed by the American College of Rheumatology (ACR) are intended to provide guidance for particular patterns of practice and not to dictate the care of a particular patient. The ACR considers adherence to these guidelines and recommendations to be voluntary, with the ultimate determination regarding their application to be made by the physician in light of each patient's individual circumstances. Guidelines and recommendations are intended to promote beneficial or desirable outcomes but cannot guarantee any specific outcome.

Understanding treatment decision making in juvenile idiopathic arthritis: a qualitative assessment.

Background: The increase in therapeutic options for juvenile idiopathic arthritis (JIA) has added complexity to treatment decisions. Shared decision making has the potential to help providers and families work together to choose the best possible option for each patient from the array of choices. As part of a needs assessment, prior to design and implementation of shared decision making interventions, we conducted a qualitative assessment of clinicians' current approaches to treatment decision making in JIA.

2013 update of the 2011 American College of Rheumatology recommendations for the treatment of juvenile idiopathic arthritis: recommendations for the medical therapy of children with systemic juvenile idiopathic arthritis and tuberculosis screening among children receiving biologic medications.

Guidelines and recommendations developed and/or endorsed by the American College of Rheumatology (ACR) are intended to provide guidance for particular patterns of practice and not to dictate the care of a particular patient. The ACR considers adherence to these guidelines and recommendations to be voluntary, with the ultimate determination regarding their application to be made by the physician in light of each patient's individual circumstances. Guidelines and recommendations are intended to promote beneficial or desirable outcomes but cannot guarantee any specific outcome.

The landscape of comparative effectiveness research in rheumatology.

Comparative effectiveness research (CER) is a growing area of investigation aimed at determining the most beneficial treatments for patients in view of their clinical characteristics. CER provides personalized treatment information that cannot be obtained from traditional clinical trials. However, many analytical challenges to performing CER remain, particularly in nonexperimental settings.

PROMIS Pediatric Peer Relationships Scale: development of a peer relationships item bank as part of social health measurement.

Objective: This study's objective was to develop a measure of social health using item response theory as part of the Patient Reported Outcomes Measurement Information System (PROMIS).

Methods: After candidate items were generated from review of prior literature, focus groups, expert input, and cognitive interviews, items were administered to youth aged 8-17 as part of the PROMIS pediatric large scale testing. Exploratory and confirmatory factor analyses were used to assess dimensionality and to identify instances of local dependence.

Upper-extremity and mobility subdomains from the Patient-Reported Outcomes Measurement Information System (PROMIS) adult physical functioning item bank.

Objective: To create upper-extremity and mobility subdomain scores from the Patient-Reported Outcomes Measurement Information System (PROMIS) physical functioning adult item bank.

Design: Expert reviews were used to identify upper-extremity and mobility items from the PROMIS item bank. Psychometric analyses were conducted to assess empirical support for scoring upper-extremity and mobility subdomains.

PROMIS® Parent Proxy Report Scales for children ages 5-7 years: an item response theory analysis of differential item functioning across age groups.

Objective: The objective of the present study is to describe the extension of the National Institutes of Health Patient-Reported Outcomes Measurement Information System (PROMIS(®)) pediatric parent proxy-report item banks for parents of children ages 5-7 years, and to investigate differential item functioning (DIF) between the data obtained from parents of 5-7-year-old children with the data obtained from parents of 8-17 year-old children in the original construction of the scales.

Methods: Item response theory (IRT) analyses of DIF were conducted comparing data from the 5-7 age group with data from the established scales for ages 8-17 across 5 generic health domains (physical functioning, pain, fatigue, emotional health, and social health) and asthma.

Results: IRT DIF analyses revealed that the majority of the items functioned similarly with responses from parents of younger and older children.

Development and psychometric properties of the PROMIS(®) pediatric fatigue item banks.

Purpose: This paper reports on the development and psychometric properties of self-reported pediatric fatigue item banks as part of the Patient-Reported Outcomes Measurement Information System (PROMIS).

Methods: Candidate items were developed by using PROMIS qualitative methodology. The resulting 39 items (25 tiredness related and 14 energy related) were field tested in a sample that included 3,048 participants aged 8-17 years.

Comparative effectiveness of nonbiologic versus biologic disease-modifying antirheumatic drugs for rheumatoid arthritis.

Objective: To evaluate the comparative effectiveness of nonbiologic disease-modifying antirheumatic drugs (DMARD) versus biologic DMARD (bDMARD) for treatment of rheumatoid arthritis (RA), using 2 common analytic approaches.

Methods: We analyzed change in Clinical Disease Activity Index (CDAI) scores in patients with RA enrolled in a US-based observational registry from 2001 to 2008 using multivariable (MV) regression and propensity score (PS) matching. Among patients who initiated treatment with a nonbiologic DMARD (n = 1729), we compared patients who switched to, or added, another nonbiologic (n = 182) or a bDMARD (n = 342) at 5, 9, and 24 months after treatment change.

Advances in Patient-Reported Outcomes: The NIH PROMIS(®) Measures.

Patient-reported outcomes (PRO) are questionnaire measures of patients' symptoms, functioning, and health-related quality of life. They are designed to provide important clinical information that generally cannot be captured with objective medical testing. In 2004, the National Institutes of Health launched a research initiative to improve the clinical research enterprise by developing state-of-the-art PROs.

Disclosure and self-report of emotional, social, and physical health in children and adolescents with chronic pain--a qualitative study of PROMIS pediatric measures.

Objectives: To examine the content validity of the Patient-Reported Outcomes Measurement Information System pediatric measures, including the pain interference scale, among children and adolescents (aged 8-18 years) who experience chronic pain. To describe children's understandings of the health domain constructs and elucidate verbal and conceptual aspects of self-reported pain-related functioning, which shape disclosure and reporting.

Methods: 34 children and youth with diagnoses of juvenile idiopathic arthritis or noninflammatory chronic pain completed semistructured and cognitive interviews exploring the meaning, experience, and expression of up to 4 of the Patient-Reported Outcomes Measurement Information System pediatric domains: anger, anxiety, depressive symptoms, fatigue, pain interference, and peer relationships.

Disease-modifying antirheumatic drug use in the treatment of juvenile idiopathic arthritis: a cross-sectional analysis of the CARRA Registry.

Objective: To characterize disease-modifying antirheumatic drug (DMARD) use for children with juvenile idiopathic arthritis (JIA) in the United States and to determine patient factors associated with medication use.

Methods: We analyzed cross-sectional baseline enrollment data from the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry from May 2010 through May 2011 for children with JIA. Current and prior medication use was included.