scholarly journals 2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Juvenile Dermatomyositis

2017 ◽  
Vol 76 (5) ◽  
pp. 782-791 ◽  
Author(s):  
Lisa G Rider ◽  
Rohit Aggarwal ◽  
Angela Pistorio ◽  
Nastaran Bayat ◽  
Brian Erman ◽  
...  
Keyword(s):  
Conjoint Analysis ◽  
Juvenile Dermatomyositis ◽  
Face Validity ◽  
Nominal Group ◽  
Response Criteria ◽  
Major Improvement ◽  
Moderate Improvement ◽  
Core Set ◽  
Improvement Score ◽  
Cent Change

To develop response criteria for juvenile dermatomyositis (DM). We analysed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial. At a consensus conference, experts considered 14 top candidate criteria based on their performance characteristics and clinical face validity, using nominal group technique. Consensus was reached for a conjoint analysis–based continuous model with a total improvement score of 0–100, using absolute per cent change in core set measures of minimal (≥30), moderate (≥45), and major (≥70) improvement. The same criteria were chosen for adult DM/polymyositis, with differing thresholds for improvement. The sensitivity and specificity were 89% and 91–98% for minimal improvement, 92–94% and 94–99% for moderate improvement, and 91–98% and 85–86% for major improvement, respectively, in juvenile DM patient cohorts using the IMACS and PRINTO core set measures. These criteria were validated in the PRINTO trial for differentiating between treatment arms for minimal and moderate improvement (p=0.009–0.057) and in the RIM trial for significantly differentiating the physician's rating for improvement (p<0.006). The response criteria for juvenile DM consisted of a conjoint analysis–based model using a continuous improvement score based on absolute per cent change in core set measures, with thresholds for minimal, moderate, and major improvement.

scholarly journals 2016 American College of Rheumatology/European League Against Rheumatism criteria for minimal, moderate, and major clinical response in adult dermatomyositis and polymyositis

2017 ◽  
Vol 76 (5) ◽  
pp. 792-801 ◽  
Author(s):  
Rohit Aggarwal ◽  
Lisa G Rider ◽  
Nicolino Ruperto ◽  
Nastaran Bayat ◽  
Brian Erman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Conjoint Analysis ◽  
Relative Weight ◽  
Nominal Group ◽  
Response Criteria ◽  
Analysis Model ◽  
Major Improvement ◽  
Core Set ◽  
Improvement Score ◽  
Cent Change

To develop response criteria for adult dermatomyositis (DM) and polymyositis (PM). Expert surveys, logistic regression, and conjoint analysis were used to develop 287 definitions using core set measures. Myositis experts rated greater improvement among multiple pairwise scenarios in conjoint analysis surveys, where different levels of improvement in 2 core set measures were presented. The PAPRIKA (Potentially All Pairwise Rankings of All Possible Alternatives) method determined the relative weights of core set measures and conjoint analysis definitions. The performance characteristics of the definitions were evaluated on patient profiles using expert consensus (gold standard) and were validated using data from a clinical trial. The nominal group technique was used to reach consensus. Consensus was reached for a conjoint analysis-based continuous model using absolute per cent change in core set measures (physician, patient, and extramuscular global activity, muscle strength, Health Assessment Questionnaire, and muscle enzyme levels). A total improvement score (range 0–100), determined by summing scores for each core set measure, was based on improvement in and relative weight of each core set measure. Thresholds for minimal, moderate, and major improvement were ≥20, ≥40, and ≥60 points in the total improvement score. The same criteria were chosen for juvenile DM, with different improvement thresholds. Sensitivity and specificity in DM/PM patient cohorts were 85% and 92%, 90% and 96%, and 92% and 98% for minimal, moderate, and major improvement, respectively. Definitions were validated in the clinical trial analysis for differentiating the physician rating of improvement (p<0.001). The response criteria for adult DM/PM consisted of the conjoint analysis model based on absolute per cent change in 6 core set measures, with thresholds for minimal, moderate, and major improvement.

Initial Development and Validation of the Global Citizenship Scale

2010 ◽  
Vol 15 (5) ◽  
pp. 445-466 ◽  
Author(s):  
Duarte B. Morais ◽  
Anthony C. Ogden
Keyword(s):  
Civic Engagement ◽  
Social Responsibility ◽  
Global Citizenship ◽  
Three Dimensional ◽  
Nominal Group Technique ◽  
Face Validity ◽  
Nominal Group ◽  
Global Competence ◽  
Confirmatory Factor Analyses ◽  
Initial Development

The purpose of this article is to report on the initial development of a theoretically grounded and empirically validated scale to measure global citizenship. The methodology employed is multi-faceted, including two expert face validity trials, extensive exploratory and confirmatory factor analyses with multiple datasets, and a series of three small-group interviews utilizing nominal group technique to verify the scope of the global citizenship construct. The findings provide support for a three-dimensional Global Citizenship Scale that encompasses social responsibility, global competence, and global civic engagement. Global competence and global civic engagement are both strong dimensions of global citizenship, and each has three reliable subdimensions that add further refinement to the construct. Social responsibility proves to be a dimension of global citizenship with a less clearly defined structure. The Global Citizenship Scale and its conceptual framework have important implications for education abroad outcomes research and practice.

Pilot Study of the Juvenile Dermatomyositis Consensus Treatment Plans: A CARRA Registry Study

2020 ◽  
Vol 48 (1) ◽  
pp. 114-122 ◽  
Author(s):  
Kuan Liu ◽  
George Tomlinson ◽  
Ann M. Reed ◽  
Adam M. Huber ◽  
Olli Saarela ◽  
...  
Keyword(s):  
Missing Data ◽  
Pilot Study ◽  
Comparative Effectiveness ◽  
Juvenile Dermatomyositis ◽  
Confounding By Indication ◽  
Childhood Arthritis ◽  
Moderate Improvement ◽  
Treatment Plans ◽  
Effectiveness Studies ◽  
New Onset

Objectives.To determine the feasibility of comparing the Childhood Arthritis and Rheumatology ResearchAlliance (CARRA) consensus treatment plans (CTP) in treating moderate new-onset juvenile dermatomyositis (JDM) using the CARRA registry, and to establish appropriate analytic methods to control for confounding by indication and missing data.Methods.A pilot cohort of 39 patients with JDM from the CARRA registry was studied. Patients were assigned by the treating physician, considering patient/family preferences, to 1 of 3 CTP: methotrexate (MTX) and prednisone (MP); intravenous (IV) methylprednisolone, MTX, and prednisone (MMP); or IV methylprednisolone, MTX, prednisone, and IV immunoglobulin (MMPI). The primary outcome was the proportion of patients achieving moderate improvement at 6 months under each CTP. Statistical methods including multiple imputation and inverse probability of treatment weighting were used to handle missing data and confounding by indication.Results.Patients received MP (n = 13), MMP (n = 18) and MMPI (n = 8). Patients in all CTP had significant improvement in disease activity. Of the 36 patients who remained in our pilot study at 6 months, 16 (44%) of them successfully achieved moderate improvement at 6 months (6/13, 46% for MP; 7/15, 47% for MMP; 3/8, 38% for MMPI). After correcting for confounding, there were no statistically significant pairwise differences between the CTP (P = 0.328–0.88).Conclusion.We gained valuable experience and insight from our pilot study that can be used to guide the design and analysis of comparative effectiveness studies using the CARRA registry CTP approach. Our analytical methods can be adopted for future comparative effectiveness studies and applied to other rare disease observational studies.

Clinical outcomes

2016 ◽  
Author(s):  
Astrid van Tubergen ◽  
Robert Landewé
Keyword(s):  
Clinical Practice ◽  
Medical Care ◽  
Clinical Outcomes ◽  
International Society ◽  
Axial Spondyloarthritis ◽  
Response Criteria ◽  
The Core ◽  
Core Set ◽  
Core Sets ◽  
Chronic Course

In general, axial spondyloarthritis (axSpA) follows a chronic course, requiring regular medical care and monitoring. The outcome of axSpA may vary substantially due to heterogenic presentation. For both research and clinical practice, it is important to have relevant, reliable, validated instruments for measuring outcome, to evaluate patients in a standardized way and capture all disease aspects. The Assessment in SpondyloArthritis international Society has developed core sets and instruments to measure these domains, and recommends only the most important domains being measured with best available methods. This chapter provides an overview of the most important outcomes in axSpA and most commonly used instruments to measure these. Additional measures frequently used but not (yet) included in the core set are addressed, and several sets of response criteria applied in axSpA research described. This chapter also provides guidance in which setting (research versus practice) and with which frequency these measures can be used.

Childhood Arthritis and Rheumatology Research Alliance Consensus Clinical Treatment Plans for Juvenile Dermatomyositis with Persistent Skin Rash

2016 ◽  
Vol 44 (1) ◽  
pp. 110-116 ◽  
Author(s):  
Adam M. Huber ◽  
Susan Kim ◽  
Ann M. Reed ◽  
Ruy Carrasco ◽  
Brian M. Feldman ◽  
...  
Keyword(s):  
Skin Rash ◽  
Complete Resolution ◽  
Juvenile Dermatomyositis ◽  
Clinical Treatment ◽  
Muscle Disease ◽  
Nominal Group ◽  
Rheumatology Research ◽  
Childhood Arthritis ◽  
Treatment Plans ◽  
Research Alliance

Objective.Juvenile dermatomyositis (JDM) is the most common form of idiopathic inflammatory myopathy in children. While outcomes are generally thought to be good, persistence of skin rash is a common problem. The goal of this study was to describe the development of clinical treatment plans (CTP) for children with JDM characterized by persistent skin rash despite complete resolution of muscle involvement.Methods.The Childhood Arthritis and Rheumatology Research Alliance, a North American consortium of pediatric rheumatologists and other healthcare providers, used a combination of Delphi surveys and nominal group consensus meetings to develop CTP that reflected consensus on typical treatments for patients with JDM with persistent skin rash.Results.Consensus was reached on patient characteristics and outcome assessment. Patients should have previously received corticosteroids and methotrexate (MTX). Three consensus treatment plans were developed. Plan A added intravenous immunoglobulin (IVIG) if it was not already being used. Plan B added mycophenolate mofetil, while Plan C added cyclosporine. Continuation of previous treatments, including corticosteroids, MTX, and IVIG, was permitted in plans B and C.Conclusion.Three consensus CTP were developed for use in children with JDM and persistent skin rash despite complete resolution of muscle disease. These CTP reflect typical treatment approaches and are not to be considered treatment recommendations or standard of care. Using prospective data collection and statistical methods to account for nonrandom treatment assignment, it is expected that these CTP will be used to allow treatment comparisons, and ultimately determine the best treatment for these patients.

scholarly journals Consensus-based recommendations for the management of juvenile dermatomyositis

2016 ◽  
Vol 76 (2) ◽  
pp. 329-340 ◽  
Author(s):  
Felicitas Bellutti Enders ◽  
Brigitte Bader-Meunier ◽  
Eileen Baildam ◽  
Tamas Constantin ◽  
Pavla Dolezalova ◽  
...  
Keyword(s):  
Rheumatic Diseases ◽  
Online Survey ◽  
Juvenile Dermatomyositis ◽  
Exercise Physiology ◽  
Nominal Group Technique ◽  
Standard Of Care ◽  
Diagnosis And Treatment ◽  
Nominal Group ◽  
Consensus Process ◽  
Treatment Pathways

BackgroundIn 2012, a European initiative called Single Hub and Access point for pediatric Rheumatology in Europe (SHARE) was launched to optimise and disseminate diagnostic and management regimens in Europe for children and young adults with rheumatic diseases. Juvenile dermatomyositis (JDM) is a rare disease within the group of paediatric rheumatic diseases (PRDs) and can lead to significant morbidity. Evidence-based guidelines are sparse and management is mostly based on physicians' experience. Consequently, treatment regimens differ throughout Europe.ObjectivesTo provide recommendations for diagnosis and treatment of JDM.MethodsRecommendations were developed by an evidence-informed consensus process using the European League Against Rheumatism standard operating procedures. A committee was constituted, consisting of 19 experienced paediatric rheumatologists and 2 experts in paediatric exercise physiology and physical therapy, mainly from Europe. Recommendations derived from a validated systematic literature review were evaluated by an online survey and subsequently discussed at two consensus meetings using nominal group technique. Recommendations were accepted if >80% agreement was reached.ResultsIn total, 7 overarching principles, 33 recommendations on diagnosis and 19 recommendations on therapy were accepted with >80% agreement among experts. Topics covered include assessment of skin, muscle and major organ involvement and suggested treatment pathways.ConclusionsThe SHARE initiative aims to identify best practices for treatment of patients suffering from PRD. Within this remit, recommendations for the diagnosis and treatment of JDM have been formulated by an evidence-informed consensus process to produce a standard of care for patients with JDM throughout Europe.

scholarly journals AGRASS Questionnaire: Assessment of Risk Management in Health Care

2020 ◽  
Vol 54 ◽  
pp. 21
Author(s):  
Zenewton André da Silva Gama ◽  
Pedro Jesus Saturno-Hernandez ◽  
Anna Claudia Sales Gomes Caldas ◽  
Marise Reis de Freitas ◽  
Ana Elza Oliveira de Mendonça ◽  
...  
Keyword(s):  
Health Care ◽  
Risk Management ◽  
High Reliability ◽  
Measurement Instrument ◽  
Face Validity ◽  
Nominal Group ◽  
Aggregated Data ◽  
Validity Analysis ◽  
Confirmatory Factor ◽  
Questionnaire Assessment

OBJECTIVE: This study aims to assess the development and the validity analysis of the Assessment of Risk Management in Health Care Questionnaire (AGRASS). METHODS: This is a validation study of a measurement instrument following the stages: 1) Development of conceptual model and items; 2) Formal multidisciplinary assessment; 3) Nominal group for validity analysis with national specialists; 4) Development of software and national pilot study in 62 Brazilian hospitals 5) Delphi for validity analysis with the users of the questionnaire. In stages 3 and 5, the items were judged based on face validity, content validity, and utility and viability, by a 1-7 Likert scale (cut-off point: median < 6). Accuracy and reliability of the questionnaire were analyzed with the Confirmatory Factor Analysis and the Cronbach’s alpha. RESULTS: The initial version of the instrument (98 items) was adapted during stages 1 to 3 for the final version with 40 items, which were considered relevant, of adequate content, useful, and viable. The instrument has 2 dimensions and 9 subdimensions, and the items have closed-ended questions (yes or no). The software for the automatic collection and analysis generates indicators, tables, and automatic graphs for the assessed institution and aggregated data. The adjustment indices confirmed a bi-dimensional model composed of structure and process (X2/gl = 1.070, RMSEA ≤ 0.05 = 0.847, TLI = 0.972), with high reliability for the AGRASS Questionnaire (α = 0.94) and process dimension (α = 0.93), and adequate for the structural dimension (α = 0.70). CONCLUSIONS: The AGRASS Questionnaire is a potentially useful instrument for the surveillance and monitoring of the risk management and patient safety in health services.

scholarly journals Development of a consensus core dataset in juvenile dermatomyositis for clinical use to inform research

2017 ◽  
Vol 77 (2) ◽  
pp. 241-250 ◽  
Author(s):  
Liza J McCann ◽  
Clarissa A Pilkington ◽  
Adam M Huber ◽  
Angelo Ravelli ◽  
Duncan Appelbe ◽  
...  
Keyword(s):  
Disease Activity ◽  
Support Groups ◽  
Juvenile Dermatomyositis ◽  
Nominal Group ◽  
Clinical Use ◽  
Web Based ◽  
Face To Face ◽  
Core Dataset ◽  
Activity Measures ◽  
Disease Damage

ObjectivesThis study aimed to develop consensus on an internationally agreed dataset for juvenile dermatomyositis (JDM), designed for clinical use, to enhance collaborative research and allow integration of data between centres.MethodsA prototype dataset was developed through a formal process that included analysing items within existing databases of patients with idiopathic inflammatory myopathies. This template was used to aid a structured multistage consensus process. Exploiting Delphi methodology, two web-based questionnaires were distributed to healthcare professionals caring for patients with JDM identified through email distribution lists of international paediatric rheumatology and myositis research groups. A separate questionnaire was sent to parents of children with JDM and patients with JDM, identified through established research networks and patient support groups. The results of these parallel processes informed a face-to-face nominal group consensus meeting of international myositis experts, tasked with defining the content of the dataset. This developed dataset was tested in routine clinical practice before review and finalisation.ResultsA dataset containing 123 items was formulated with an accompanying glossary. Demographic and diagnostic data are contained within form A collected at baseline visit only, disease activity measures are included within form B collected at every visit and disease damage items within form C collected at baseline and annual visits thereafter.ConclusionsThrough a robust international process, a consensus dataset for JDM has been formulated that can capture disease activity and damage over time. This dataset can be incorporated into national and international collaborative efforts, including existing clinical research databases.

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