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

    liza.mccann@alderhey.nhs.uk

  • 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