Reliability of Functional Outcome Measures in Neurofibromatosis 1

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Brief Title

Reliability of Functional Outcome Measures in Neurofibromatosis 1

Official Title

A Study Investigating the Inter- and Intra- Rater Reliability of a Battery of Functional Standardised Outcome Measures in Neurofibromatosis 1

Brief Summary

      Neurofibromatosis 1 (NF1) is a common inherited condition that affects the skin, bone and
      nervous system. The complications of NF1 are widespread and can lead to varied difficulties
      dependent on the location of neurofibromas and their secondary complications. At present
      evaluation of the effect of treatments in NF1 is based on a clinician's neurological
      assessment, magnetic resonance imaging and positron emission tomography but it is apparent
      that imaging results do not always link with clinical and functional changes in an
      individual. This study aims to evaluate the reliability (stability) of a range of functional
      standardised outcome measures in adults with NF1.
    

Detailed Description

      Neurofibromatosis 1 (NF1) is an inherited condition that occurs in 1 in 2,500 births;
      affecting the skin, bone and nervous system. The complications of NF1 are widespread and can
      lead to varied difficulties dependent on the location of neurofibromas and their secondary
      complications. One person with NF1 may have minimal issues whereas another may be profoundly
      disabled; one may have difficulties using their hands and arms and another may have
      difficulties standing from a chair. New pharmacological and nonpharmacological treatments for
      NF1 are currently being developed and they have potential to dramatically influence a person
      with NF1's life. At present evaluation of treatment effect in NF1 is based on a clinician's
      neurological assessment, magnetic resonance imaging and positron emission tomography and it
      is apparent that imaging results do not always link with clinical and functional changes in
      an individual. Therefore clinicians and researchers agree that the analysis of treatment
      effect needs to include the evaluation of a person's function. The findings from this study
      will improve the care of people with NF1 by ensuring that we are able to measure functional
      changes in a person with NF1 with greater confidence, meaning that we can start, continue or
      cease treatments based on the holistic picture of an individual.

      The world confederation of physiotherapists (WCPT) stipulates that standardised outcome
      measures (SOM) are used to provide a quantitative measure of capability, and to evaluate the
      impact of treatment on function. There are a wide range of SOM's that evaluate function (e.g.
      walking, standing from a chair, use of hands) so a SOM is chosen by the clinician based on
      the patient's presenting functional difficulty and the scientific credibility of the SOM.
      Scientific credibility (robustness) is based on scientific properties of the SOM i.e. how
      stable the SOM is (reliability) and whether it assesses what it is intending to assess
      (validity). It is important to evaluate each SOM within the target population as variability
      within that condition, strongly influences the SOM's reliability and validity (de Vel 2011).

      At present, functional SOM's have limited scientific and subsequent clinical credibility when
      used in people with NF1 because their scientific properties have not yet been evaluated
      within this patient group which means that if they are used clinically or within research,
      there is an inherent element of doubt when interpreting the SOM's results. The objective of
      this research team is to create a core set of functional SOM's which have undergone rigorous
      scientific evaluation so that they can be used in the future for clinical practice and for
      research purposes in adults with NF1.

      This study aims to evaluate four commonly used SOM's for reliability in adults with NF1 (over
      16 years of age). The measures chosen for evaluation are the 10 metre walk test, the timed up
      and go test, the functional reach test and the nine hole peg test. Interrater reliability
      (the test result remains the same when reviewed by different people) and intrarater
      reliability (the test result remains stable when reviewed twice by the same tester) will be
      evaluated. From this data, we will be able to calculate the standard error of measurement an
      additional important scientific characteristics of SOM evaluation.
    


Study Type

Observational


Primary Outcome

10 metre walk test


Condition

Neurofibromatosis 1, NF1


Study Arms / Comparison Groups

 Outcome measurement
Description:  Each participant will be asked to complete each standardised outcome measure (SOM) three times and each trial will be videotaped by the researcher. The selected SOM's are the 10 metre walk test, the timed up and go test, the functional reach test and the nine-hole peg test. A physiotherapist will watch the video on 2 separate occasions to evaluate intra-rater reliability. Inter-rater reliability will be assessed through asking three other neurofibromatosis specialist professionals (two NF1 consultants and one NF1 specialist nurse) to review the video and to score each measure completed. Once the filmed sessions have been analysed by the relevant clinician's the data will be destroyed in line with Trust policy.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

49

Start Date

June 2015

Completion Date

September 2015

Primary Completion Date

September 2015

Eligibility Criteria

        Inclusion Criteria:

          -  All patients aged 16 years or older who fulfil the diagnostic criteria for NF1 who do
             not have a significant mobility or balance impairment that is unrelated to their NF1,

          -  Able to walk more than 10 metres without physical assistance (may use walking aids)

          -  Patients who attend the national NF1 service at GSTT

        Exclusion Criteria:

          -  Patients who are unable to provide informed consent will be unable to participate in
             this study.
      

Gender

All

Ages

16 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations


NCT ID

NCT02479360

Organization ID

RJ115/N174


Responsible Party

Sponsor

Study Sponsor

Guy's and St Thomas' NHS Foundation Trust


Study Sponsor

, , 


Verification Date

June 2015