Benedetta Schiappapietra

A New Approach to Clinical Care of Juvenile Idiopathic Arthritis: The Juvenile Arthritis Multidimensional Assessment Report

Objective. To develop and test a new multidimensional questionnaire for assessment of children with juvenile idiopathic arthritis (JIA) in standard clinical care. Methods. The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) includes 15 parent or patient-centered measures or items that assess well-being, pain, functional status, health-related quality of life, morning stiffness, disease activity, disease status and course, joint disease, extraarticular symptoms, side effects of medications, therapeutic compliance, and satisfaction with illness outcome. The JAMAR is proposed for use as both a proxy-report and a patient self-report, with the suggested age range of 7–18 years for use as a self-report. From March 2007 to September 2009, the questionnaire was completed by the parents of 618 children with JIA in 1814 visits and by 332 children in 749 visits. Results. The JAMAR was found to be feasible and to possess face and content validity. All parents and children reported that the questionnaire was simple and easy to understand. Completion and scoring appeared to be quick, requiring < 15 minutes. There were very few missing data. Parents’ proxy-reported and children’s self-reported data were remarkably concordant. The JAMAR provided thorough information for the study patients about recent medical history and current health status. It performed similarly across different children’s ages and characterized the level of disease activity and disability well. Conclusion. The development of the JAMAR introduces a new approach in pediatric rheumatology practice. This new questionnaire may help enhance the quality of care of children with JIA.

Parent and child acceptable symptom state in juvenile idiopathic arthritis.

Objective. To explore the parent and child acceptable symptom state in juvenile arthritis (JA-PASS and JA-CASS, respectively) and estimate the JA-PASS and JA-CASS cutoff values for outcome measures. Methods. Children with juvenile idiopathic arthritis (JIA) and their parents completed a multi-dimensional questionnaire that included parent-reported and child-reported outcomes and a question about whether they considered the disease state as satisfactory. Additional assessments included demographic data, physician-reported outcomes, and acute-phase reactant levels. Stepwise logistic regression was used to assess contributors to JA-PASS and JA-CASS. Cutoff values of outcome measures that defined JA-PASS and JA-CASS were determined using both 75th percentile and receiver-operating characteristic (ROC) curve methods. Testing procedures included evaluation of discriminative and construct validity of the satisfaction question and assessment of reliability of JA-PASS and JA-PASS cutoffs. Results. Of 584 parents, 385 (65.9%) considered their child in JA-PASS. Of 343 children, 236 (68.8%) considered themselves in JA-CASS. Significant contributors to being in either JA-PASS or JA-CASS were absence of active joints, better rating of overall well-being, and better physical function or health. Cutoff values yielded by 75th percentile and ROC curve methods were similar. Parent, child, and physician global ratings yielded the lowest percentage of false-positive misclassification and the best tradeoff between sensitivity and specificity. The satisfaction question showed good discriminative and construct validity and the JA-PASS and JA-PASS cutoffs were found to be stable over time. Conclusion. The acceptable symptom state is a relevant concept for children with JIA and their parents and constitutes a valid outcome measure that is potentially applicable in routine practice and clinical trials.

A new short and simple health-related quality of life measurement for paediatric rheumatic diseases: initial validation in juvenile idiopathic arthritis

OBJECTIVE To develop and validate a new short and simple measure of health-related quality of life (HRQL) in children with juvenile idiopathic arthritis (JIA). METHODS The Paediatric Rheumatology Quality of Life Scale (PRQL) is a 10-item questionnaire that explores HRQL in two domains: physical health (PhH) and psychosocial health (PsH). Validation of the parent proxy report and child self-report versions of the instrument was accomplished by evaluating 472 JIA patients and approximately 800 healthy children. Validation analyses included assessment of feasibility, face and content validity; construct and discriminative ability; internal structure and consistency; test-retest reliability; responsiveness to clinical change; and minimal clinically important difference. RESULTS The PRQL was found to be feasible and to possess both face and content validity. The PRQL score correlated in the predicted range with most of the other JIA outcome measures, thereby demonstrating good construct validity, and discriminated well between different levels of disease severity. Assessment of internal structure (factor analysis) revealed that the PhH and PsH subscales identify two unambiguously separated domains. The internal consistency (Cronbachs alpha) was 0.86. The intraclass correlation coefficient for test-retest reliability was 0.91. The PRQL revealed fair responsiveness, with a standardized response mean of 0.67 in improved patients. Overall, the PRQL appeared to be more able to capture physical HRQL than psychosocial HRQL. CONCLUSION The PRQL was found to possess good measurement properties and is, therefore, a valid instrument for the assessment of HRQL in children with JIA. This tool is primarily proposed for use in standard clinical care.

Defining Criteria for Disease Activity States in Nonsystemic Juvenile Idiopathic Arthritis Based on a Three-Variable Juvenile Arthritis Disease Activity Score

To determine cutoff values for defining the states of inactive disease (ID), low disease activity (LDA; or minimal disease activity), moderate disease activity (MDA), and high disease activity (HDA) using the clinical (3‐variable) Juvenile Arthritis Disease Activity Score (cJADAS).

Clinical outcome measures in juvenile idiopathic arthritis.

Juvenile idiopathic arthritis (JIA), as a chronic condition, is associated with significant disease- and treatment-related morbidity, thus impacting children’s quality of life. In order to optimize JIA management, the paediatric rheumatologist has begun to regularly use measurements of disease activity developed, validated and endorsed by international paediatric rheumatology professional societies in an effort to monitor the disease course over time and assess the efficacy of therapeutic interventions in JIA patients.A literature review was performed to describe the main outcome measures currently used in JIA patients to determine disease activity status.The Juvenile Disease Activity Score (JADAS), in its different versions (classic JADAS, JADAS-CRP and cJADAS) and the validated definitions of disease activity and response to treatment represent an important tool for the assessment of clinically relevant changes in disease activity, leading more and more to a treat-to-target strategy, based on a tight and thorough control of the patient condition. Moreover, in recent years, increasing attention on the incorporation of patient-reported or parent-reported outcomes (PRCOs), when measuring the health state of patients with paediatric rheumatic diseases has emerged.We think that the care of JIA patients cannot be possible without taking into account clinical outcome measures and, in this regard, further work is required.

Preliminary validation of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR) in 403 clinic patients

Methods The JAMAR includes: Juvenile Arthritis Functionality Scale (JAFS) (0 = normal; 30 = worst), Pediatric Rheumatology Quality of Life (PRQL) questionnaire (0 = best; 30 = worst); visual analogue scales (0 = best; 10 = worst) for parent/patient rating of well-being (WB), pain (P), disease activity (DA); parent/patient assessment of morning stiffness, disease status, satisfaction about disease outcome.

Chapter 7 – Juvenile Idiopathic Arthritis

Juvenile idiopathic arthritis (JIA) is a heterogeneous condition and represents the most common rheumatologic disease in childhood. A variety of cardiac manifestations have been described in JIA. Pericarditis and myocarditis are the most common systemic subset and cardiac failure is a life-threatening complication of macrophage activation syndrome. Valvular heart disease, most frequently aortic insufficiency, has occasionally been seen in rheumatoid factor-positive polyarthritis and juvenile ankylosing spondylitis. The significance of the observed impairment in systolic and diastolic ventricle function is unclear as JIA is not associated with increased prevalence of ischemic heart disease or cardiomyopathy. The risk of premature atherosclerosis is also uncertain. Although some studies have shown endothelial and vascular dysfunction, the carotid intima-media thickness was not found to be consistently thicker than in healthy controls. In spite of decreased anaerobic fitness, the exercise capacity of children with chronic arthritis is preserved irrespective of the stage of disease activity.

PReS-FINAL-2176: Declines in levels of disease activity and physical disability in children with juvenile idiopathic arthritis seen in standard clinical care over the last 25 years

Over the last 3 decades there have been important advances in the management of juvenile idiopathic arthritis (JIA), which include the introduction of methotrexate and, later on, the tendency toward its earlier initiation, the widespread use of intra-articular corticosteroid injections, and, more recently, the availability of the biologic response modifiers. Although this therapeutic progress is likely to have led to a marked improvement in the outlook of children with JIA, the prognostic impact of the newer therapeutic modalities is still poorly documented.

Agreement between objective and subjective definitions of inactive disease, remission and minimal disease activity in juvenile idiopathic arthritis

Background In juvenile idiopathic arthritis (JIA), substantial disagreement between physicians, parents and children over disease remission can lead to difficulty in assessing the efficacy of treatments. It is, therefore, important to ascertain whether clinicians’, parents’ and children’s opinions converge and diverge and whether the recently developed criteria for inactive disease (ID) or minimal disease activity (MDA) may help enhance concordance. Objective

Comparison of physician's, parent's and patient's global ratings made on linear or 21-circle visual analogue scales (VAS) in juvenile idiopathic arthritis (JIA)

15th Paediatric Rheumatology European Society (PreS) Congress Wietse Kuis, Patricia Woo, Angelo Ravelli, Hermann Girschick, Michael Hofer, Johannes Roth, Rotraud K Saurenmann, Alberto Martini, Pavla Dolezova, Janjaap van der Net, Pierre Quartier, Lucy Wedderburn and Jan Scott Meeting abstracts – A single PDF containing all abstracts in this Supplement is available here .