Treatment outcomes in 63 cases of juvenile dermatomyositis-associated calcinosis.

Objectives: We performed a multi-institutional retrospective review of patients treated for juvenile dermatomyositis (JDM)-associated calcinosis to analyse the association between treatment outcomes and patient, disease, and treatment characteristics.

Methods: Childhood Arthritis and Rheumatology Research Alliance investigators searched their electronic health records for patients with JDM and calcinosis treated between 2003 and 2019 and analysed data at JDM diagnosis, calcinosis diagnosis, and calcinosis treatment. Statistical methods included univariable and multivariable analyses, Kaplan-Meier estimates, and multivariable Cox models.

Remission and low disease activity definitions in adult idiopathic inflammatory myopathies: A narrative review by myositis clinical trials consortium (MCTC).

Idiopathic inflammatory myopathies (IIM) are a heterogeneous group of rare systemic autoimmune rheumatic diseases. Despite advances in treatment, the definition of remission and low disease activity (LDA) in IIM remains inconsistent and lacks consensus and validation. This review summarizes existing published definitions, achievement rates, and predictive factors of remission/LDA in adult IIM, focusing on dermatomyositis (DM), polymyositis (PM), anti-synthetase syndrome (ASyS), and immune-mediated necrotizing myopathies (IMNM).

CAR T cell therapy for children with rheumatic disease: the time is now.

Initial success with B cell-targeted chimeric antigen receptor (CAR) T cells for the treatment of systemic lupus erythematosus and other rheumatic diseases has generated enthusiasm for the broad application of this technology outside of the field of oncology. Paediatric patients with severe rheumatic diseases require lifelong therapy with a substantial toxicity burden and a high cost of care. Paradigm-shifting treatments, including CAR T cells, are desperately needed.

Psychometric properties of patient-reported outcomes measurement information system (PROMIS) fixed short forms in Juvenile Myositis.

Objectives: Assess reliability and validity of Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric self-report and parent-proxy report fixed short forms in juvenile myositis (JM).

Methods: Children with JM (8-17yo) and parents of 5-17 yo JM patients completed PROMIS measures (Physical Function, Pain Interference, Fatigue, Emotional Distress), PedsQL Generic Core scales and Rheumatology Module (PedsQL-GC/-RM). Internal consistency reliability was assessed via Cronbach's alpha.

Approach to Janus kinase inhibition for juvenile dermatomyositis among CARRA and PReS providers.

Objectives: Janus kinase inhibition (JAKi) has been proposed as a treatment for idiopathic inflammatory myopathies to target increased interferon signalling. Predominantly retrospective reports have demonstrated effectiveness of JAKi in refractory JDM. However, JAKi remains an off-label treatment for JDM and there may be variation in use worldwide.

Longitudinal program evaluation of an inter-institutional mentorship network for pediatric rheumatology using a quality improvement framework.

Background: The American College of Rheumatology (ACR)/Childhood Arthritis and Rheumatology Research Alliance (CARRA) Mentoring Interest Group (AMIGO) is an inter-institutional mentorship program launched to target mentorship gaps within pediatric rheumatology. Initial program evaluation indicated increased mentorship access. Given the small size of the pediatric rheumatology workforce, maintaining a consistent supply of mentors was a potential threat to the longevity of the network.

Mental health in paediatric and adult myositis-related diseases: current state of research, interventions, and future steps from the MIHRA Psychological Impact Scientific Working Group.

Psychological and emotional well-being are critical aspects of overall health for individuals with chronic rheumatologic conditions. Mental health-related literature, however, predominantly focuses on systemic lupus erythematosus or rheumatoid arthritis, with limited emphasis on idiopathic inflammatory myopathies (IIMs). High proportions of those with juvenile myositis report psychological distress at levels warranting mental health referral.

Longitudinal program evaluation of an inter-institutional mentorship network for pediatric rheumatology using a quality improvement framework.

Background: The American College of Rheumatology (ACR)/Childhood Arthritis and Rheumatology Research Alliance (CARRA) Mentoring Interest Group (AMIGO) is an inter-institutional mentorship program launched to target mentorship gaps within pediatric rheumatology. Initial program evaluation indicated increased mentorship access. Given the small size of the pediatric rheumatology workforce, maintaining a consistent supply of mentors was a potential threat to the longevity of the network.

The impact of the COVID-19 pandemic on pediatric rheumatology practice: an international, cross-sectional survey study.

Objective: The COVID-19 pandemic has affected patient care in general. We aimed to analyze the impact of the pandemic on pediatric rheumatology practice.

Methods: An online survey including 22 questions was created by the representatives of the Emerging RheumatoloGists and rEsearchers (EMERGE) group of the Pediatric Rheumatology European Society (PReS) on SurveyMonkey.

The Effect of Psychiatric Comorbidity on Healthcare Utilization for Youth With Newly Diagnosed Systemic Lupus Erythematosus.

Objective: To examine the effect of psychiatric diagnoses on healthcare use in youth with systemic lupus erythematosus (SLE) during their first year of SLE care.

Methods: We conducted a retrospective cohort study using claims from 2000 to 2013 from Clinformatics Data Mart (OptumInsight). Youth aged 10 years to 24 years with an incident diagnosis of SLE (≥ 3 International Classification of Diseases, 9th revision, codes for SLE 710.

Baseline characteristics of children with juvenile dermatomyositis enrolled in the first year of the new Childhood Arthritis and Rheumatology Research Alliance registry.

Background: To report baseline characteristics, patient reported outcomes and treatment of children with Juvenile Dermatomyositis (JDM) in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry.

Methods: Children newly diagnosed with JDM were enrolled in the CARRA Registry from 41 pediatric rheumatology centers. Baseline patient demographics, disease characteristics, assessments, patient reported outcome and treatments were recorded.

2021 American College of Rheumatology/Vasculitis Foundation Guideline for the Management of Kawasaki Disease.

Objective: To provide evidence-based recommendations and expert guidance for the management of Kawasaki disease (KD), focusing on clinical scenarios more commonly addressed by rheumatologists.

Methods: Sixteen clinical questions regarding diagnostic testing, treatment, and management of KD were developed in the Patient/Population, Intervention, Comparison, and Outcomes (PICO) question format. Systematic literature reviews were conducted for each PICO question.

2021 American College of Rheumatology/Vasculitis Foundation Guideline for the Management of Kawasaki Disease.

Objective: To provide evidence-based recommendations and expert guidance for the management of Kawasaki disease (KD), focusing on clinical scenarios more commonly addressed by rheumatologists.

Methods: Sixteen clinical questions regarding diagnostic testing, treatment, and management of KD were developed in the Patient/Population, Intervention, Comparison, and Outcomes (PICO) question format. Systematic literature reviews were conducted for each PICO question.

Cardiovascular Health in Pediatric Rheumatologic Diseases.

Cardiovascular disease risk is evident during childhood for patients with juvenile systemic lupus erythematosus, juvenile dermatomyositis, and juvenile idiopathic arthritis. The American Heart Association defines cardiovascular health as a positive health construct reflecting the sum of protective factors against cardiovascular disease. Disease-related factors such as chronic inflammation and endothelial dysfunction increase cardiovascular disease risk directly and through bidirectional relationships with poor cardiovascular health factors.

Comparison of Patient-Reported Outcomes Measurement Information System Computerized Adaptive Testing Versus Fixed Short Forms in Juvenile Myositis.

Objective: Patient-Reported Outcomes Measurement Information System (PROMIS) measures can be administered via computerized adaptive testing (CAT) or fixed short forms (FSFs), but the empirical benefits of CAT versus FSFs are unknown in juvenile myositis (JM). The present study was undertaken to assess whether PROMIS CAT is feasible, precise, correlated with FSFs, and less prone to respondent burden and floor/ceiling effects than FSFs in JM.

Methods: Patients 8-17 years of age (self-report and parent proxy) and parents of patients 5-7 years of age (only parent proxy) completed PROMIS fatigue, pain interference, upper extremity function, mobility, anxiety, and depressive symptoms measures.

Utility of patient-reported outcomes measurement information system (PROMIS) physical function form in inflammatory myopathy.

Objective: Idiopathic inflammatory myopathies (IIM) are a group of diseases characterized by muscle weakness, which limit activities of daily living. Patient Reported Outcomes Measurement Information System (PROMIS) is a set of outcome measures developed using NIH funding, but has not yet been studied in adult IIM. Currently, the most commonly used PROs in IIM are Health Assessment Questionnaire (HAQ-DI) and SF-36 physical function-10 (PF10), both of which have several limitations.

Engaging patients and parents to improve mental health intervention for youth with rheumatological disease.

Background: Mental health disorders are common in youth with rheumatological disease yet optimal intervention strategies are understudied in this population. We examined patient and parent perspectives on mental health intervention for youth with rheumatological disease.

Methods: We conducted a mixed methods cross-sectional study, via anonymous online survey, developed by researchers together with patient/parent partners, to quantitatively and qualitatively examine youth experiences with mental health services and resources in North America.

Parent Perspectives on Addressing Emotional Health for Children and Young Adults With Juvenile Myositis.

Objective: To assess parent perspectives regarding the emotional health impact of juvenile myositis (JM) on patients and families, and to assess preferences for emotional health screening and interventions.

Methods: Parents of children and young adults with JM were purposively sampled for participation in focus groups at the Cure JM Foundation National Family Conference in 2018. Groups were stratified by patient age group (6-12, 13-17, and 18-21 years), and conversations were audiorecorded, transcribed verbatim, and co-coded via content analysis, with subanalysis by age group.

The importance of development standards for anchoring vignettes: an illustrative example from pediatric localized scleroderma quality of life.

Purpose: Anchoring vignettes (AVs) are a promising measurement technique to reduce bias in patient-reported outcome (PRO) measures by helping researchers understand differences in how individuals and groups interpret response options. However, little attention has been paid to ensure quality development of AVs, and their performance has not been well assessed in pediatric populations. In this study, we explore the application of a rigorous development process for AVs based upon current standards for PROs, as well as feasibility of AVs when administered to children and adolescents.

Adverse Childhood Experiences Are Associated with Childhood-Onset Arthritis in a National Sample of US Youth: An Analysis of the 2016 National Survey of Children's Health.

Objectives: To determine whether there is an association between adverse childhood experiences (ACEs) and childhood-onset arthritis, comparing youth with arthritis to both healthy youth and youth with other acquired chronic physical diseases (OCPD); and to examine whether ACEs are associated with disease-related characteristics among children with arthritis.

Study Design: In a cross-sectional analysis of data from the 2016 National Survey of Children's Health we examined whether ACEs were associated with having arthritis vs either being healthy or having a nonrheumatologic OCPD. ACE scores were categorized as 0, 1, 2-3, ≥4 ACEs.