Development of an Internationally agreed Minimal Dataset for JDM
Research type
Research Study
Full title
Development of an Internationally agreed Minimal Dataset for Juvenile Dermatomyositis (JDM) for clinical and research use.
IRAS ID
160667
Contact name
Liza McCann
Contact email
Sponsor organisation
Alder Hey Children’s NHS Foundation Trust
Clinicaltrials.gov Identifier
IPHS-1314-321 , UoL ethics approval
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
Aim:
Gain international agreement on which clinical features / tests are most important for clinical and research use in juvenile dermatomyositis (JDM), forming a ‘minimal dataset’.Why it is important:
Juvenile dermatomyositis (JDM) is a severe, potentially life-threatening disease. Being rare, affecting 2-4 per million children per year, international collaboration is crucial to improve outlook. An internationally agreed minimal dataset has the potential to unify on-going data collections, aid communication, provide a standard of care, raise awareness, evaluate treatments or patient/parent reported outcome measures and help to answer questions like ‘how patients develop the disease’, ‘which tests are useful for diagnosis’ or ‘which features suggest severe disease’. It could provide a framework for future clinical trials.Study design:
Part 1A - Delphi survey: Two consecutive web-based surveys will be distributed to >500 healthcare professionals via membership of established organisations across UK, Europe, USA and Canada. Participants are asked to rank importance of each variable for clinical care and research.
Part 1B - Patient / parent involvement: Patients / parents will be asked which outcomes they consider important by completing a questionnaire in clinic or by completing an electronic questionnaire on-line. Specific questions will also be addressed by groups of young people / parents in the UK.
Part 2 - Consensus meeting using nominal group technique: Informed by the above, an international panel of experts will determine through a structured process during a 2-day face-to-face meeting, which items should be part of a minimal dataset.
Part 3 - Testing the dataset in real-life practice: The minimal dataset will be tested via established patient databases. After 6-months data-collection, secondary anonymised data will be analysed by our group for rate of completion / accuracy. The final minimal dataset will be endorsed through existing collaborations.Funding: ARUK (reference 2017): £48,789.27 (3 years).
REC name
East Midlands - Nottingham 1 Research Ethics Committee
REC reference
14/EM/1259
Date of REC Opinion
14 Nov 2014
REC opinion
Favourable Opinion