Core Outcome Measures in Effectiveness Trials

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

General Information

Abstract:
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.

This is an Open Access article distributed in accordance with the terms of the Creative Commons Attribution (CC BY 4.0) license, which permits others to distribute, remix, adapt and build upon this work, for commercial use, provided the original work is properly cited. See: http://creativecommons.org/licenses/by/4.0/

http://dx.doi.org/10.1136/annrheumdis-2017-212141

Aim:
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.

Authors:
Dr Liza McCann (PI)
Department of Rheumatology, Alder Hey Children's Hospital, University of Liverpool

Professor Michael Beresford (co-applicant)
Institute of Translational Medicine, University of Liverpool

Dr Jamie Kirkham (co-applicant)
Department of Biostatistics, University of Liverpool

Dr Clarissa Pilkington (co-applicant)
Department of Paediatric Rheumatology, University College London

Liza J McCann, Clarissa A Pilkington, Adam M Huber, Angelo Ravelli, Duncan Appelbe, Jamie J Kirkham, Paula R Williamson, Amita Aggarwal, Lisa Christopher-Stine, Tamas Constantin, Brian M Feldman, Ingrid Lundberg, Sue Maillard, Pernille Mathiesen, Ruth Murphy, Lauren M Pachman, Ann M Reed, Lisa G Rider, Annet van Royen-Kerkof, Ricardo Russo, Stefan Spinty, Lucy R Wedderburn, Michael W Beresford

Professor Lucy Wedderburn (co-applicant)
Institute of Child Health, University College London

Publication

Journal:
Ann Rheum Dis
Volume:
0
Issue:
Pages:
1 - 10
Year:
2017
DOI:
Further Study Information

Date:
January 2014 - 2016
Funding source(s):
Arthritis Research UK

Health Area

Disease Category
Child health
Rheumatology

Disease Name
Juvenile dermatomyositis (JDM)
Myositis

Target Population

Age Range
-

Sex
Unknown


Nature / type of Intervention
Unknown

Method(s)

Delphi process
Nominal group technique (NGT)

A provisional dataset, formulated through structured processes by a representative group of JDM experts will be scrutinised by modified Delphi exercises, using electronic software, distributed via international organisations. This will help inform discussion during a meeting of JDM experts, who will determine, by nominal group consensus, feasibility, validity, acceptability, format, definition and glossary of variables within the proposed minimal dataset. The dataset will be tested within existing databases before finalisation.

Representatives from Paediatric Rheumatology and Myositis expert groups (internationally) including IMACS, PReS JDM working party, PRINTO, UK JDCBS, Euromyositis, CARRA and OMERACT, along with lay representatives and dermatology colleagues will be involved in the consensus process.


Stakeholders Involved

Clinical experts
Consumers (caregivers)
Consumers (patients)

Study Type

Minimum dataset

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