Development and Validation of the GCA PRO questionnaire
Research type
Research Study
Full title
Development and validation of a patient reported outcome measure for Giant Cell Arteritis
IRAS ID
263802
Contact name
Joanna C Robson
Contact email
Sponsor organisation
University Hospitals NHS Foundation Trust
Duration of Study in the UK
1 years, 11 months, 30 days
Research summary
Research Summary:
Aims:
To develop a Patient Reported Outcome Measure (PRO) for Giant Cell Arteritis (GCA), in the form of a questionnaire specific to the disease and its impact on quality of life. The aim is for the questionnaire to be used in clinical trials to measure health-related quality of life of greatest importance to patients.
Background:
GCA causes inflamed blood vessels in the head and neck and affects one or two persons per 10,000 people in the UK.
In our interviews, patients with GCA have described their quality of life:
“one day I just couldn’t get out the bed, I couldn't bend my knees or walk. “ 73 year-old female.“it was a bit like watching a very cheap DVD on the television. When it all goes crackle, crackle, crackle and breaks up, that’s how my vision was.” 74 year-old male.
“they used to call me Super Nan, but they can’t call me that now.” 75 year-old female.
Patients receive high dosages of steroids, which have side-effects including weight gain, anxiety, high blood pressure and diabetes. Newer targeted treatments are only available to a few patients.
Design and methods
We have conducted 31 interviews and identified areas of interest to patients. Based on the interviews, we have co-developed with patients 40 questions which are now ready for testing. We will recruit 500-1000 patients with GCA confirmed by a clinician. Screening of participants via Rheumatology and Ophthalmology sites (approx. 40-50 sites). We will ask patients to complete the 40 questions at baseline and 3 days later. We will collect data on the patient's disease and background. The analysis will involve refining the questionnaire by removing unnecessary questions and ensure it is a robust measure for quality of life.
Two patient partners co-applicants, including a trustee of PMRGCAUK, are actively involved (e.g. reviewing protocols including recruitment methods and patient documentation and interpretation of results).
Summary of Results:
The GCA PRO study team is led by the University of the West of England Bristol and University of Leeds and includes two patient partners who have GCA. The team have been developing a Patient Reported Outcome Measure (PROM) for Giant Cell Arteritis (GCA). This is a questionnaire which measures how GCA and its treatment impacts patients' health-related quality of life.
The questionnaire has been designed to be used in clinical trials and clinical practice to measure aspects of greatest importance to patients with GCA and to help patients and their clinicians communicate with each other.
Firstly, we interviewed patients with GCA from the UK and Australia about their experiences of GCA. We took care to include people with a range of different GCA presentations (e.g. those with and without cranial disease, visual involvement and large vessel vasculitis. The analysis of this interview data identified a range of important topics related to GCA itself, and how it impacted on daily life. This work was published in the journal Rheumatology in January 2021 and has also formed the basis of questions for the PROM, based on the topics identified.
Once we had drafted an initial PROM questionnaire, we carried out further patient interviews to test it. This process is called cognitive testing and it helped to refine the format and wording. The questionnaire was also checked to ensure it will be easy to translate into other languages in the future. The final result is a PROM with 40 questions related to the patient perception of GCA and its treatment.
During 2019 and 2020 we performed a large-scale patient testing of the GCA-PRO. We worked with clinicians and research teams to recruit patients from rheumatology and ophthalmology departments across 38 NHS sites.
The tremendous efforts and hard work of site teams has meant that in spite of the very challenging circumstances of the last year, we exceeded our recruitment target figure of 400. We received a total of 427 completed sets of questionnaires from patients. The data from these questionnaires helped us refine the questionnaire by removing unnecessary questions and to ensure it is a consistent and reliable measure for use in clinical trials and practice. The overall length has also been reduced to 30 questions to make it more user friendly.
For the final phase of the study, we looked at how the PROM works in practice. 10-20 pairs of patients and clinicians were recruited in Bristol/Weston-Super-Mare and Leeds. Each patient was supported to complete the questionnaire before their clinical appointment. It was then used as a communication tool during their consultation. We got feedback from both patients and clinicians after the appointment about how the use of the PRO helped discussion between patients and clinicians, and may aid in shared decision making.REC name
South Central - Oxford A Research Ethics Committee
REC reference
19/SC/0439
Date of REC Opinion
10 Oct 2019
REC opinion
Further Information Favourable Opinion