Neurofibromatosis 2 Outcome Measure Study

  • Research type

    Research Study

  • Full title

    A study investigating the inter rater and intra rater reliability of a variety of functional outcome measures in adults with Neurofibromatosis 2.

  • IRAS ID

    200912

  • Contact name

    Rebecca Smith

  • Contact email

    Rebecca.smith@gstt.nhs.uk

  • Sponsor organisation

    Guy's and St Thomas' NHS Foundation Trust

  • Duration of Study in the UK

    0 years, 6 months, 1 days

  • Research summary

    Neurofibromatosis 2 (NF2) is a condition which affects approximately one in 33 000 births. Those with NF 2 usually present with tumours (schwannomas) in their inner ear, or vestibular organ, with common symptoms including problems with hearing, balance, dizziness and vision.\n\nThe complications of NF 2 can lead to varied difficulties with activities of daily living and may impair quality of life. More recently, new pharmacological treatments for NF2 are being trialled which may have the potential to dramatically influence quality of life in those with NF 2. Currently, evaluation of treatment effect in NF2 is based on clinician’s neurological assessment and imaging. However, imaging results do not always correlate with clinical and functional changes of the individual. Therefore, evaluating the effect of interventions or treatment should include the evaluation of a person’s function, using standardised outcome measures.\n\nThere are a wide range of SOM’s that evaluate function, and therefore a SOM is chosen based on the patient’s presenting functional difficulty and the scientific credibility of the measure. The credibility of the measure or robustness is based on scientific evaluation of the SOM in the disease population, i.e. how stable the SOM is (reliability) and whether it assesses what it is intending to assess (validity). At present, functional SOM’s have limited scientific and subsequent clinical trustworthiness when used in people with NF2, because their properties have not yet been evaluated within this population. \n\nTherefore, this study aims to evaluate the reliability (stability) of different SOMs in adults with NF2. Interrater reliability (the test result remaining the same when reviewed by different people) and intrarater reliability (the test result remaining stable when reviewed twice by the same tester) will be evaluated. We would then be able to calculate the standard error of measurement and minimal clinical detectable change; important characteristics in SOM evaluation.

  • REC name

    North West - Liverpool Central Research Ethics Committee

  • REC reference

    16/NW/0504

  • Date of REC Opinion

    21 Jun 2016

  • REC opinion

    Favourable Opinion