Objectives This 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.
Methods A 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.
Results A 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.
Conclusions Through 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.
- patient perspective
- outcomes research
- autoimmune diseases
- multidisciplinary team-care
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Handling editor Tore K Kvien
Contributors LJM has led all parts of the study including background work, preparation of the protocol, ethics submissions, content of surveys, planning of consensus meeting, testing the dataset and writing the manuscript. CAP, AMH, AR and LRW, as members of the steering committee, have provided intellectual input and practical help into all parts of the study including background work, protocol development, Delphi survey, planning of the consensus meeting, reviewing results, refining the dataset and preparing the manuscript. AMH, AR and CAP also tested the dataset in practice. DA developed the bespoke Delphi system and provided IT support for the study including data analysis. JJK participated in the study design, was responsible for testing the Delphi system, performed the statistical analysis, helped prepare for the consensus meeting, and was involved in reviewing results and preparing the manuscript. PRW has provided expert advice on study design and Delphi methodology and analysis. AA, LC-S, TC, BMF, IL, SM, PM, RM, LMP, AMR, LGR, AvRK, RR and SS attended the consensus meeting and had intellectual input into the study. In addition, AA, AvRK, LMP, LGR and RR tested the dataset in practice. MWB has been responsible for intellectual and financial overview of the study, input into the protocol development and as a member of the steering committee has provided intellectual input into the Delphi survey and consensus meeting, reviewing results, facilitating the consensus meeting, refining the dataset and preparing the manuscript.
Funding This work was supported by Arthritis Research UK (grant number 20417); January 2014–July 2017. The UK JDM Cohort and Biomarker study has been supported by generous grants from the Wellcome Trust UK (085860), Action Medical Research UK (SP4252), The Myositis Support Group UK, Arthritis Research UK (14518), The Henry Smith Charity. LRW’s work is supported in part by Great Ormond Street Children’s Charity, the GOSH/ICH NIHR funded Biomedical Research Centre (BRC) and Arthritis Research UK. The JDM Cohort study is adopted onto the Comprehensive Research Network through the Medicines for Children Research Network (www.mcrn.org.uk) and is supported by the GOSH/ICH Biomedical Research Centre. LGR was supported in part by the Intramural Research Program of the National Institute of Environmental Health Sciences National Institutes of Health.
Competing interests IEL has research grants from Astra Zeneca and Bristol Myers Squibb and has served on advisory board for Bristol Myers Squibb.
Patient consent Obtained.
Ethics approval Ethical approval for the study was obtained from the IPHS Research Ethics Committee (reference IPHS-1314-321). The NHS Research Ethics Committee approved final questionnaires, consent/assent forms and patient/parent information leaflets (NRES Committee East Midlands, reference 14/EM/1259; IRAS project ID 160667).
Provenance and peer review Not commissioned; externally peer reviewed.
Data sharing statement All data for this study are held securely within the University of Liverpool. There are no unpublished data openly available. A URL has been established at Liverpool University to hold the published dataset forms as described within the manuscript.
Collaborators International Myositis Assessment and Clinical Studies group (IMACS), Paediatric Rheumatology INternational Trials Organisation (PRINTO at www.printo.it), Juvenile Dermatomyositis Research Group (JDRG) UK and Ireland, Childhood Arthritis and Rheumatology Research Alliance (CARRA), Paediatric Rheumatology European Society (PReS) JDM working group, Euromyositis Steering Committee, Core Outcome Measures in Effectiveness Trials (COMET) and Outcome Measures in Rheumatology (OMERACT) groups. A full list of contributors to the JDCBS can be found on the JDRG website (http://www.juveniledermatomyositis.org.uk).
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