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Objective: Patients with juvenile‐onset systemic lupus erythematosus (JSLE) have increased atherosclerosis risk. This study investigated novel atherosclerosis progression biomarkers in the Atherosclerosis Prevention in Pediatric Lupus Erythematosus (APPLE) trial, the largest investigator‐led randomized control trial of atorvastatin versus placebo for atherosclerosis progression in JSLE, using carotid intima‐media thickness (CIMT) as the primary outcome. Methods: Unsupervised clustering of baseline CIMT and CIMT progression over 36 months was used to stratify patients with JSLE. Disease characteristics, cardiovascular risk scores, and baseline serum metabolome were investigated in CIMT‐stratified patients. Machine learning techniques were used to identify and validate a serum metabolomic signature of CIMT progression. Results: Baseline CIMT stratified patients with JSLE (N = 151) into three groups with distinct high, intermediate, and low CIMT trajectories irrespective of treatment allocation, despite most patients having low cardiovascular disease risk based on recommended assessment criteria. In the placebo group (n = 60), patients with high versus low CIMT progression had higher total (P = 0.001) and low‐density lipoprotein (LDL) (P = 0.002) cholesterol levels, although within the reference range. Furthermore, a robust baseline metabolomic signature predictive of high CIMT progression was identified in the placebo arm (area under the curve, 80.7%). Patients treated with atorvastatin (n = 61) had reduced LDL cholesterol levels after 36 months, as expected; however, despite this, 36% still had high atherosclerosis progression, which was not predicted by metabolomic biomarkers, suggesting nonlipid drivers of atherosclerosis in JSLE with management implications for this subset of patients. Conclusion: Significant baseline heterogeneity and distinct subclinical atherosclerosis progression trajectories exist in JSLE. Metabolomic signatures can predict atherosclerosis progression in some patients with JSLE with relevance for clinical trial stratification. [ABSTRACT FROM AUTHOR]

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.Results: A total of 483 childhood-onset SLE patients were assessed. Care for the 310 US patients differed markedly for childhood-onset SLE-QIs addressing LN, bone health, vaccinations, education on CV risk, and transition planning. Performance of safety blood testing for medications was high at all centers. Despite often similar performance on the childhood-onset SLE-QI, access to kidney biopsies was lower in Brazil than in the US. Irrespective of the country of practice, larger centers tended to meet the childhood-onset SLE-QIs more often than smaller centers.Conclusion: The childhood-onset SLE-QIs, evidence-based minimum standards of medical care, are not consistently met in the US or some other countries outside the US. This has the potential to contribute to suboptimal childhood-onset SLE outcomes. [ABSTRACT FROM AUTHOR]

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. For item bank development, we identified existing items from measures in the literature, grouped them by concept, removed redundancies, and modified the remaining items to match PROMIS formatting. New items were written as needed and cognitive debriefing was completed with the children and their parents, resulting in 98 pain behavior (47 self, 51 proxy), 54 quality, and 4 intensity items for further testing. Qualitative content analyses suggest that reportable pain outcomes that matter to children with pain are captured within and consistent with the pain domain framework in PROMIS.Perspective: PROMIS pediatric pain behavior, quality, and intensity items were developed based on a theoretical framework of pain that was evaluated by multiple stakeholders in the measurement of pediatric pain, including researchers, clinicians, and children with pain and their parents, and the appropriateness of the framework was verified. [ABSTRACT FROM AUTHOR]

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. We used confirmatory factor analysis (CFA) to evaluate dimensionality and differential item functioning (DIF) analysis to evaluate parameter stability between genders and by age; we examined residual correlations to evaluate local dependence (LD) among items and estimated the parameters of item response theory (IRT) models. Results: Of 3,048 participants, 48 % were males, 60 % were white, and 23 % had at least one chronic condition. CFA results suggest two moderately correlated factors. Two items were removed due to high LD, and three due to gender-based DIF. Two item banks were calibrated separately using IRT: Tired and (Lack of) Energy, which consisted of 23 and 11 items, respectively; 10- and 8-item short-forms were created. Conclusion: The PROMIS assessment of self-reported fatigue in pediatrics includes two item banks: Tired and (Lack of) Energy. Both demonstrated satisfactory psychometric properties and can be used for research settings. [ABSTRACT FROM AUTHOR]

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. Items that met the unidimensionality criteria were subsequently calibrated using Samejima's Graded Response Model. Differential item functioning was examined by gender and age. Results: The sample included 3,048 youth who completed the questionnaire (51.8% female, 60% white, and 22.7% with chronic illness). The initial conceptualization of social function and sociability did not yield unidimensional item banks. Rather, factor analysis revealed dimensions contrasting peer relationships and adult relationships. The analysis also identified dimensions formed by responses to positively versus negatively worded items. The resulting 15-item bank measures quality of peer relationships and has strong psychometric characteristics as a full bank or an 8-item short form. Conclusions: The PROMIS pediatric peer relationships scale demonstrates good psychometric characteristics and addresses an important aspect of child health. [ABSTRACT FROM AUTHOR]

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. Methods: Pediatric rheumatology clinicians were recruited from 2 academic children's hospitals affiliated with a quality improvement learning network, using purposive and snowball sampling. Semi-structured interviews elicited how clinicians with prescribing authority (n = 10) interact with families to make treatment decisions. Interviews were audio-recorded and transcribed verbatim. A multi-disciplinary research team used content analysis to analyze the interview data. To validate data from individual interviews and enrich our understanding, we presented the interview results to pediatric rheumatology clinicians attending a learning network meeting (n = 24 from 12 children's hospitals). We then asked the clinicians questions to further identify and discuss areas of variation in the decision-making processes. Results: Clinicians described a decision-making process in which they, rather than the family or other care team members, consistently initiated treatment decisions. Initial treatment options presented to families generally reflected the clinician's preferred treatment approaches, which differed across clinicians. Clinicians used various methods to inform families about treatment options and tailor information according to perceptions of a family's information needs, level of comprehension or mood (e.g. anxiety). The attributes of medication presented to families fell into 4 categories: benefits, risks, logistics and family preferences. Clinicians typically included family members in the decision to initiate JIA treatment after limiting the options to fit the clinical situation and the clinician's own preferences. Family members' preferences were seen as more integral in the decision to stop treatment after symptom remission. Conclusions: Decision making about initial JIA treatment appears to be largely driven by clinician preferences. Family preferences are more likely to be considered for treatment discontinuation. Opportunities exist to develop, test, and implement tools to facilitate shared decision making in pediatric rheumatology. [ABSTRACT FROM AUTHOR]

Objective There is wide variation in therapeutic approaches to systemic juvenile idiopathic arthritis (JIA) among North American rheumatologists. Understanding the comparative effectiveness of the diverse therapeutic options available for treatment of systemic JIA can result in better health outcomes. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed consensus treatment plans and standardized assessment schedules for use in clinical practice to facilitate such studies. Methods Case-based surveys were administered to CARRA members to identify prevailing treatments for new-onset systemic JIA. A 2-day consensus conference in April 2010 employed modified nominal group technique to formulate preliminary treatment plans and determine important data elements for collection. Followup surveys were employed to refine the plans and assess clinical acceptability. Results The initial case-based survey identified significant variability among current treatment approaches for new-onset systemic JIA, underscoring the utility of standardized plans to evaluate comparative effectiveness. We developed 4 consensus treatment plans for the first 9 months of therapy, as well as case definitions and clinical and laboratory monitoring schedules. The 4 treatment regimens included glucocorticoids only, or therapy with methotrexate, anakinra, or tocilizumab, with or without glucocorticoids. This approach was approved by >78% of the CARRA membership. Conclusion Four standardized treatment plans were developed for new-onset systemic JIA. Coupled with data collection at defined intervals, use of these treatment plans will create the opportunity to evaluate comparative effectiveness in an observational setting to optimize initial management of systemic JIA. [ABSTRACT FROM AUTHOR]

Objective The ability to assess quality of care is a necessary component of continuous quality improvement. The assessment typically is accomplished by determination of compliance with a defined set of quality measures (QMs). The objective of this effort was to establish a set of QMs for the assessment of the process of care in juvenile idiopathic arthritis (JIA). Methods A 12-member working group composed of representatives from the American College of Rheumatology, American Academy of Pediatrics, American Board of Pediatrics, and Association of Rheumatology Health Professionals was assembled to guide the project. Delphi questionnaires were sent to 237 health professionals involved in the care of children with JIA. A total of 471 items in 23 domains were identified. The working group met via 4 live e-meetings during which results from the Delphi questionnaires were distilled to a reduced draft set. Each working group member selected a proposed QM to investigate and present evidence from the literature as to its attributes and appropriateness for inclusion into the set. Nominal group technique was used to come to consensus on a proposed set of QMs. Results The proposed set contains 12 QMs within 4 health care domains. Each QM consists of a statement of 1) the assessment to be completed, 2) when the first assessment should be completed and a suggested frequency of assessment during followup, 3) recommendations of appropriate tools or methods of assessment, and 4) initial performance goals. Conclusion Implementation of the proposed QM set will improve the process of care, facilitate continuous quality improvement, and eventuate in improved health outcomes of children with JIA. [ABSTRACT FROM AUTHOR]

Background. Recently, the National Institutes of Health Roadmap for Medical Research initiative led a large-scale effort to develop the Patient-Reported Outcomes Measurement Information System (PROMIS). PROMIS's main goal was to develop a set of item banks and computerized adaptive tests for the clinical research community. Asthma, as the most common chronic childhood disease, was chosen for a disease-specific pediatric item bank. Objectives. The primary objective of this research is to present the details of the psychometric analyses of the asthma domain items. Methods. Item response theory (IRT) analyses were conducted on a 34–asthma item bank. Test forms containing PROMIS Pediatric Asthma domain items were completed by 622 children ages 8 to 12. Items were subsequently evaluated for local dependence, scale dimensionality, and differential item functioning. Results. A 17-item pool and an 8-item short form for the new PROMIS Pediatric Asthma Impact Scale (PAIS) were generated using IRT. The recommended 8-item short form contains the item set that provides the maximum test information at the mean (50) on the T-score metric. If more score precision is required, the complete 17-item pool is recommended and may be used in toto or as the basis of a computerized adaptive test (CAT). A shorter test form can also be created and scored on the same scale. Conclusions. The present study presents the PROMIS Pediatric Asthma Impact Scale (PAIS) developed with IRT, and provides the initial calibration data for the items. [ABSTRACT FROM AUTHOR]

Abstract: Objective: The aim of this study was to identify factors associated with the initiation of biologic agents for the treatment of rheumatoid arthritis (RA) in a large US observational cohort. Methods: Semiannual patient-reported data in the ARAMIS (Arthritis, Rheumatism and Aging Medical Information System) data bank from January 1998 to January 2006 were analyzed retrospectively using pooled logistic regression (with adjustment for center-level and temporal effects) to identify patient-, disease-, and treatment-related characteristics associated with the initiation of biologics for the treatment of RA. Results: The analysis included 1545 patients from 7 US centers. By 2006, 41.4% of 679 patients remaining in the sample had received biologics. Initiation of biologics was significantly associated with greater disability in the previous 6-month period (per 1-unit increase in Health Assessment Questionnaire score: odds ratio [OR] = 1.45; 95% CI, 1.22–1.72; P < 0.01) and treatment in the previous period with steroids (OR = 2.24; 95% CI, 1.76–2.85; P < 0.01) or nonbiologic disease-modifying antirheumatic drugs (OR = 2.43; 95% CI, 1.71–3.46; P < 0.01). Two sociodemographic factors were significant predictors of decreased use of biologics: older age (per 10 years: OR = 0.74; 95% CI, 0.660.82; P < 0.01) and lower annual income (per $10,000 reduction: OR = 0.95; 95% CI, 0.91–1.00; P = 0.04). There were no significant differences with respect to sex, race, employment status, comorbidity, previous NSAID use, or treatment center. Conclusions: Disease- and treatment-related factors were significant predictors of the initiation of biologics for RA. Independent of these factors, however, biologics were less often used in patients who were older and those with lower incomes. Use of biologics increased steadily over the period studied. [Copyright &y& Elsevier]

The article looks into the medical coverage of tumor necrosis factor alpha inhibitors as an influential factor on the prescribing behavior of physicians. Significantly, the application of etanercept or infliximab on the treatment of rheumatoid arthritis has been analyzed. Furthermore, the analyses of patient demographic and disease characteristics were conducted to characterize predictors of the biologic drug prescribed. The author points out that differential drug coverage has an impact on patient care and health care costs.

Background/Purpose: Medication options for juvenile idiopathic arthritis (JIA) are increasing. Medications differ on a variety of attributes, including mechanisms of action, dosing intervals, modes of administration, safety profiles, and cost. Some parents of children with JIA are left with questions and concerns about medications suggesting a need for improved clinician-parent communication. Decisions like this, with multiple reasonable options that differ in ways that matter to families, are conducive to 'Shared Decision Making' (SDM). SDM is a process whereby clinicians share information about the options and patients/parents share information about their goals and preferences. Together, a treatment plan is developed that is the best fit for the individual patient and their family. We conducted a project to develop tools to facilitate SDM within the Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN). PR-COIN is a learning network designed to improve outcomes of JIA care using quality improvement (QI) approaches. Methods: Development of issue cards to facilitate SDM involved multiple steps including qualitative interviews with clinicians and care providers, direct observation of clinical encounters, and an iterative design process involving a graphic designer and a stakeholder panel of clinicians, allied health professionals and parents. We introduced the prototype issue cards for use in clinics using Plan-Do-Study-Act (PDSA) cycles. We elicited feedback to guide card revisions from PR-COIN members via weekly electronic surveys and discussions during monthly webinars and following direct observation of encounters using the cards. Results: We identified the medication attributes deemed most important to discuss by our stakeholders, including 'How Soon' will therapies take effect, 'How Often' are they given, 'Side Effects', 'Cost', 'How Long' will need to stay on the medication, and 'Other Considerations.' We used these attributes to organize the issue cards and symbols to convey key concepts (see Figure). Intended use of the cards involves clinicians showing the cards to patient and parent and asking which card they would like to discuss first. By selecting a card, the family reveals what is important to them. After 18 revisions, PR-COIN stakeholders at 6 sites found the issue cards to be acceptable for regular use. [ABSTRACT FROM AUTHOR]

Background/Purpose: The Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN) is a multi-center, quality-improvement-focused learning network with a mission of improving outcomes for children and adolescents with juvenile idiopathic arthritis (JIA). PR-COIN has developed a population management tool which facilitates pro-active, coordinated health care communications and interventions that can be used in managing JIA patient populations. Methods: The population management tool was developed with information technology expertise to link seamlessly to the PR-COIN longitudinal registry. Within the population management tool, on demand reports can be generated using participating site or aggregate, network level data according to demographics, clinical features, disease activity levels, patient reported outcomes (functional ability/CHAQ score, arthritis related pain), medication use, and quality indicators. Drill down capability enables identification of patients. Results: Currently, data from 8 sites, 1161 patients, and 5334 clinical encounters are included in the population management tool (Table ). Individual site usage of the population management tool is variable ranging from infrequent use to regular use at monthly population management meetings. The PR-COIN network is currently developing strategies to promote consistent usage of the tool at sites. Clinicians routinely using information provided through this tool report satisfaction with its functionality and ease of use; furthermore they report enhanced ability to proactively identify and address specific patient needs prior to and during clinic visits, optimizing care. [ABSTRACT FROM AUTHOR]

Background/Purpose: The NIH Patient Reported Outcomes Measurement Information System (PROMIS) has created publicly available patient reported outcomes measures in several domains of physical, social and emotional health. Measurement of pediatric pain in PROMIS is currently limited to pain interference. Pain behaviors are observable actions orreactions that communicate pain including verbal, nonverbal and pain reducing behaviors. Numerous validated parent/provider-rating scales of pain behavior in children exist but there are currently no validated self-report measures of pain behavior in school-age children and adolescents. Such measures could be useful in establishing targets for treatment and assessing outcomes. The aim of this study is to enhance PROMIS pediatric pain assessment by developing and testing pediatric pain behavior item banks for self- and proxy-report. Methods: Candidate items were developed through a qualitative item review process, and were in the format, 'In the past 7 days, when I was in pain ...' Patients ages 8 to 17 years, or parents/guardians of children, with a chronic painful condition (fibromyalgia, juvenile idiopathic arthritis, sickle cell disease) were recruited through outpatient clinics at 3 centers. Child participants completed approximately 100 PROMIS items concerning their pain (including 47 candidate pain behavior items), physical function, fatigue, and psychosocial well/being. Proxies responded to sociodemographic and health history items and 51 new candidate proxy-report pain behavior items were collected. A confirmatory factor analysis (CFA) was performed on the child and guardian pain behavior data, with model fit assessed by the comparative fit index (CFI) and root mean square error of approximation (RMSEA). Item response theory (IRT) analysis was performed on the pain behavior items based on the graded response model. Differential item functioning (DIF) was assessed by age group and disease group. Results: 450 children (71% female, Mage 13.54), and 232 proxies participated. CFA indicated unidimensionality in the child (CFI = 0.962; RMSEA = 0.079) and proxy pain behavior responses (CFI = 0.970; RMSEA = 0.080). The responses for the child and proxy data had good IRT model fit and were free of local dependence. Slopes for the pediatric responses ranged from 1.81 ('rubbed body where hurt') to 4.40 ('moved slower'), and thresholds ranged from −2.00 to 4.95. For the proxy data, slopes ranged from 1.51 ('think of something fun') to 3.48 ('tried not to move'), and thresholds ranged from −3.02 to 2.43. Items performed well across disease groups and age. There was little DIF either by age group (8-12, 13-18) or by sample (child vs. proxy). Child and proxy scores were correlated at 0.70. Correlations between pain behavior and pain intensity were 0.60 in children and 0.65 in proxy sample. Conclusion: The PROMIS pediatric pain behavior item-banks for self and proxy report are suitable for use in non-adaptive format as short forms or in dynamic format as computerized adaptive tests in clinical research with the potential for adoption into clinical care. [ABSTRACT FROM AUTHOR]

Background/Purpose: The Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN) is a multi-site quality improvement learning network that aims to improve outcomes for children and adolescents with juvenile idiopathic arthritis. In addition to addressing variations in care practices, PR-COIN has incorporated guidance and tangible contributions from patients and families to its national organizational and governance structure to drive improvement in JIA outcomes. PR-COIN aims to grow this initiative to bring care providers, patients and family members together as co-producers in improving care and outcomes. Methods: Various methods supporting family engagement are employed. PR-COIN teams invite interested parents at their respective clinical sites to bring the parent perspective and priorities into local improvement work. To assure a reliable structure, a parent engagement specialist was employed to develop a platform for bringing families and care providers together. National conference calls facilitated by the specialist establish relationships across site representatives and begin goal setting for this group. Parents contribute to shared decision making project efforts. PR-COIN's public website encourages family awareness and involvement in the network's improvement efforts. Results: 7 of 11 sites have welcomed parent contributions to their improvement efforts (e.g. focus groups, advisory activities, etc.). Parents and patients have participated in PR-COIN's biannual conferences, sharing the family perspective and needs associated with JIA care. One parent brings a unique background and skillset as both a pediatrician with QI experience and the parent of a teen with JIA. This distinction prompted appointment to PR-COIN's governing body. We incorporate patient perspectives into clinic process improvements (e.g. pain management, optimizing physical functioning), enhanced and refined PR-COIN's Shared Decision Making tool, supporting stronger, more informed medication choice, and are developing best practices optimizing use of these materials in clinic. Finally our engaged parents are encouraging others to contribute ideas supportive of PR-COIN structure, growth, strategy and improvement efforts. Conclusion: Parents and patients bring unique talents, insight and skills to the operation of quality improvement networks making efforts more relevant to the self-identified needs of JIA families. [ABSTRACT FROM AUTHOR]