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Juvenile dermatomyositis, the most common inflammatory myopathy of childhood, is a rare systemic autoimmune vasculopathy that is characterised by weakness in proximal muscles and pathognomonic skin rashes. The length of time before the initiation of treatment affects presenting symptoms, laboratory measures, and pathophysiology. It also affects disease outcomes, including the development of pathological calcifications, which are associated with increased morbidity. Both genetic and environmental risk factors seem to have a role in the cause of juvenile dermatomyositis; HLA B8–DRB1*0301 ancestral haplotype is a strong immunogenetic risk factor, and antecedent infections and birth seasonality suggest that environmental stimuli might increase risk. Activation of dendritic cells with upregulation of genes induced by type-1 interferon (α) in muscle and peripheral blood seems to be central to disease pathogenesis. Treatment often includes combinations of corticosteroids, methotrexate, and other immunosuppressive agents. Disease outcome, if treatment is initiated early, is generally good. Randomised controlled trials are needed to define the most effective treatments.

The Hemophilia Joint Health Score (HJHS) was developed and validated to detect arthropathy in children. Additional evidence is required to show validity in adults. We studied the convergent and discriminant construct validity of the HJHS version 2.1(HJHSv2.1) in adults with hemophilia. A secondary aim was to define age‐related normative adult HJHSv2.1 reference values. We studied 192 adults with hemophilia, and 120 healthy adults in four age‐matched groups—18 to 29, 30 to 40, 41 to 50, and >50 years—at nine centers. Trained physiotherapists scored the HJHS and World Federation of Hemophilia (WFH) joint score. Health history, the Functional Independence Scale of Hemophilia (FISH), Hemophilia Activities List (HAL), and Short‐Form McGill Pain Questionnaire (SF‐MPQ) were also collected. The median age was 35.0 years. Of participants with hemophilia, 68% had severe, 14% moderate, and 18% mild disease. The HJHS correlated strongly with WFH score (Spearman’s rho [rs] = .95, P < .001). Moderate correlations were seen between the FISH (rs = .50, P < .001) and SF‐MPQ Present Pain Intensity (rs = .50, P < .001), while a modest correlation was found with the HAL (rs = −.37, P < .001). The HJHS significantly differentiated between age groups (Kruskal‐Wallis T = 35.02, P < .001) and disease severity in participants with hemophilia. The HJHS had high internal reliability (Cronbach’s α = .88). We identified duration of swelling as a redundant item in the HJHS. The HJHS shows evidence of strong convergent and discriminant construct validity to detect arthropathy in adults with hemophilia and is well suited for use in this population.

Background Patient reported outcome measures (PROMs) provide valuable insight on patients’ well-being and facilitates communication between healthcare providers and their patients. The increased integration of the technology within the healthcare setting presents the opportunity to collect PROMs electronically, rather than on paper. The Childhood Health Assessment Questionnaire (CHAQ) and Quality of My Life (QoML) are common PROMs collected from pediatric rheumatology patients. The objectives of this study are to (a) determine the equivalence of the paper and electronic forms (e-form) of CHAQ and QoML questionnaires; (b) identify potential benefits and barriers associated with using an e-form to capture PROMs; and (c) gather feedback on user experience. Methods Participants completed both a paper and an e-form of the questionnaires in a randomized order, following which they completed a feedback survey. Agreement of the scores between the forms were statistically analyzed using the intraclass correlation coefficient (ICC) (95 % Confidence Interval (CI)) and bias was assessed using a Bland-Altman plot. Completion and processing times of the forms were compared using mean and median measures. Quantitative analysis was performed to assess user experience ratings, while comments were qualitatively analyzed to identify important themes. Results 196 patients participated in this project. Scores on the forms had high ICC agreement > 0.9. New patients took longer than returning patients to complete the forms. Overall, the e-form was completed and processed in a shorter amount of time than the paper form. 83 % of survey respondents indicated that they either preferred the e-form or had no preference. Approximately 10 % of respondents suggested improvements to improve the user interface. Conclusions E-forms collect comparable information in an efficient manner to paper forms. Given that patients and caregivers indicated they preferred completing PROMs in this manner, we will implement their suggested changes and incorporate e-forms as standard practice for PROMs collection in our pediatric rheumatology clinic.

Background Resilience has been shown to be associated with better psychological outcomes and ability to cope with negative and traumatic events in the healthcare setting. Therefore, in this study, we aimed to evaluate resilience and its association with disease activity and health-related quality of life (HRQOL) in children with Systemic Lupus Erythematosus (SLE) and Juvenile Idiopathic Arthritis (JIA). Findings Patients with diagnoses of SLE or JIA were recruited. We collected: demographic data, medical history and physical examination, physician and patient global health assessments, Patient Reported Outcome Measurement Information System questionnaires, Connor Davidson Resilience Scale 10 (CD-RISC 10), Systemic Lupus Erythematosus Disease Activity Index, and clinical Juvenile Arthritis Disease Activity Score 10. Descriptive statistics were calculated, and PROMIS raw scores were converted to T-scores. Spearman’s correlations were performed, with statistical significance set to p < 0.05. 47 study subjects were recruited. The average CD-RISC 10 score in SLE was 24.4, and in JIA was 25.2. In children with SLE, CD-RISC 10 was correlated with disease activity and inversely correlated with anxiety. In children with JIA, resilience was inversely associated with fatigue, and positively correlated with mobility and peer relationships. Conclusions In children with SLE and JIA, resilience is lower than in the general population. Further, our results suggest that interventions to increase resilience may improve the HRQOL of children with rheumatic disease. Ongoing study of the importance of resilience in this population, as well as interventions to increase resilience, will be an important area of future research in children with SLE and JIA.

Bayesian analysis can incorporate clinicians' beliefs about treatment effectiveness into models that estimate treatment effects. Many elicitation methods are available, but it is unclear if any confer advantages based on principles of measurement science. We review belief-elicitation methods for Bayesian analysis and determine if any of them had an incremental value over the others based on its validity, reliability, and responsiveness. A systematic review was performed. MEDLINE, EMBASE, CINAHL, Health and Psychosocial Instruments, Current Index to Statistics, MathSciNet, and Zentralblatt Math were searched using the terms (prior OR prior probability distribution) AND (beliefs OR elicitation) AND (Bayes OR Bayesian). Studies were evaluated on: design, question stem, response options, analysis, consideration of validity, reliability, and responsiveness. We identified 33 studies describing methods for elicitation in a Bayesian context. Elicitation occurred in cross-sectional studies ( n = 30, 89%), to derive point estimates with individual-level variation ( n = 19; 58%). Although 64% ( n = 21) considered validity, 24% ( n = 8) reliability, 12% ( n = 4) responsiveness of the elicitation methods, only 12% ( n = 4) formally tested validity, 6% ( n = 2) tested reliability, and none tested responsiveness. We have summarized methods of belief elicitation for Bayesian priors. The validity, reliability, and responsiveness of elicitation methods have been infrequently evaluated. Until comparative studies are performed, strategies to reduce the effects of bias on the elicitation should be used.

The Hemophilia Joint Health Score (HJHS) is a validated outcome tool developed for the assessment of joint health in people with hemophilia. The ordinal joint score assesses 9 items in 6 index joints. It is recognized as an optimal measurement of arthropathy in children and young adults. The aim of this study was to develop an updated scoring system for the HJHS that may overcome the limitations of its current ordinal scoring structure. A survey was developed using 1000Minds decision‐making software. Respondents were provided with discrete choice tasks of ranking alternatives to determine the preference weight, or relative importance, placed on different criteria for each HJHS item. The survey was distributed to an anonymous sample of health care professionals with extensive experience in the physical examination of joints in people with hemophilia. A total of 64 musculoskeletal health care professionals participated; with a 64% survey completion rate. The HJHS item weights provide a sum to 1.0; the highest‐ranked item was extension loss (0.139) followed by swelling (0.121), whereas the lowest was duration of swelling (0.057) followed by muscle atrophy (0.08). Compared to the original, the relative efficiency of the new score was 5.4. Observed differences in preference weights for HJHS items highlight the potential under‐ or overestimation of true joint health using the current ordinal scoring system. An updated scoring system using weighted items may improve the precision of HJHS assessment, leading to improved clinical management of joint health, while providing a robust research tool.

Background Juvenile Dermatomyositis (JDM) is a rare, chronic, and life-threatening childhood autoimmune disease. Currently, there are recommended, reliable and validated measurement tools for assessment of skin disease activity in JDM including the Disease Activity Score (skinDAS), Cutaneous Assessment Tool (CAT), and the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI). The Physician’s global assessment skin visual analog scale (Skin VAS) is also widely used for skin activity in JDM. For the purpose of comparative international studies, we wanted to compare these tools to the Physician’s skin VAS (as a standard) to identify which performs better. Objectives We sought to compare the correlations of these scoring tools, and separately assess the responsiveness each tool demonstrates following patient treatment, in order to see if one tool may be preferred. This was determined by assessing how well these tools correlate with each other, and the Physician’s skin VAS over time, as well as the responsiveness of each tool after patient treatment. Methods Skin scores were recorded at a baseline (first visit after June 1 st , 2018) and all follow-up office visits at the Juvenile Dermatomyositis Clinic. Following baseline visits, patients were followed up as clinically indicated. A subset of newly diagnosed patients (inception cohort) was identified. Correlations were assessed at the baseline visit and over time for the whole cohort. The correlations over time were derived using Generalized Estimating Equations (GEEs). Standardized response means with 95% confidence intervals were calculated to test score responsiveness for the nested inception cohort. Results The skinDAS, CAT and CDASI all correlated highly with each other and with the Physician’s skin VAS. The three scoring tools accurately reflected Physician’s skin VAS scores over time. In addition, all tools showed moderate to high responsiveness following treatment. Conclusion All studied skin score tools performed well in our study and appear to be useful. Since no tool far outperforms the others, arbitrary consensus will be needed to select a single standard measurement tool for the purposes of efficiency and global comparability.

The goal of this study was to determine the difference in participant understanding, satisfaction, timing and, preference between video consent and written informed consent in a pediatric rheumatology research setting. Participants were randomized to receive either video consent or written informed consent for a registry study. After completing the first consent method, they completed a comprehension and satisfaction questionnaire. Then they received the alternate consent method and completed a second set of questionnaires. Bayesian non-parametric tests determined the difference in comprehension, satisfaction, timing and preference between video consent and written informed consent. Ninety-nine caregivers and 76 patients were randomized into video consent (n = 88) and written informed consent (n = 87) groups. Comprehension (Max = 12) and satisfaction (Max = 5) were high in both groups. There was moderate evidence supporting no difference in comprehension (median video consent  = 11 and median written informed consent  = 10) and satisfaction (median video consent  = 4 and median written informed consent  = 5) between video consent and written informed consent (BF 10  = 0.225 and 0.32, respectively). The median time to complete video consent and written informed consent was 408 (95% Credible Interval (CrI): 397–412) and 360 (95% CrI: 329–391) seconds, respectively. There was decisive evidence that video consent increased the time of consent (in our sample by 48 seconds) compared to written informed consent (BF 10  = 713). There was decisive evidence for participants preferring video consent over written informed consent (BF 10  = 2.307x10 11 ) as they thought it was easier to follow. Overall, participant understanding and satisfaction were comparable between video consent and written informed consent. Even though video consent was slightly less time efficient compared to written informed consent, video consent was highly preferred by caregivers and patients, supporting its use to obtain informed consent.

To investigate how consensus is operationalized in Delphi studies and to explore the role of consensus in determining the results of these studies. Systematic review of a random sample of 100 English language Delphi studies, from two large multidisciplinary databases [ISI Web of Science (Thompson Reuters, New York, NY) and Scopus (Elsevier, Amsterdam, NL)], published between 2000 and 2009. About 98 of the Delphi studies purported to assess consensus, although a definition for consensus was only provided in 72 of the studies (64 a priori). The most common definition for consensus was percent agreement (25 studies), with 75% being the median threshold to define consensus. Although the authors concluded in 86 of the studies that consensus was achieved, consensus was only specified a priori (with a threshold value) in 42 of these studies. Achievement of consensus was related to the decision to stop the Delphi study in only 23 studies, with 70 studies terminating after a specified number of rounds. Although consensus generally is felt to be of primary importance to the Delphi process, definitions of consensus vary widely and are poorly reported. Improved criteria for reporting of methods of Delphi studies are required.

Background Juvenile Dermatomyositis (JDM) is a pediatric vasculopathy characterized primarily by skin and muscle involvement. Cardiac findings have been reported in children with JDM but have rarely been investigated in detail. Methods We aimed to describe the relevant clinical and laboratory cardiac findings of a cohort of patients with JDM, followed at one centre, at disease diagnosis. Results We performed a retrospective review of 105 patients with JDM, followed from 1991 to 2007. Six of 70 patients (9%, 6% of the entire cohort) had abnormal electrocardiographic (ECG) findings, while 26 of 54 patients (48%, 25% of the entire cohort) had abnormal echocardiographic (echo) findings. Many of these findings were either mild or unlikely to be a result of JDM. Conclusions Our findings suggest that cardiac abnormalities at JDM disease onset are frequently seen, but are rarely significant findings due to disease; however, JDM patients should be considered for screening for cardiac disease as late cardiac complications are well recognized.