Psychometric evaluation of a Patient Reported Symptom Index for NMIBC
Research type
Research Study
Full title
NMIBC-SI Evaluation Psychometric evaluation of a Patient-Reported Symptom Index for Non-Muscle Invasive Bladder Cancer: Field Testing
IRAS ID
235841
Contact name
Euan Green
Contact email
Sponsor organisation
Salford Royal NHS Foundation Trust
Duration of Study in the UK
2 years, 4 months, 31 days
Research summary
Research Summary
The primary goal in managing patients with Non muscle invasive bladder cancer is to completely remove the tumour and control the unpredictable risk of recurrence and progression to muscle invasion with as little treatment burden and side effects as possible.A PRO(patient reported outcome) is any report coming directly from patients, without interpretation by physicians or others, about how they feel in relation to a health condition and its therapy. PROs can include symptoms, function, health-related quality of life (HRQoL), and perceptions of treatment. These patient-reports are captured and quantified by PRO measures (PROMs) in the form of questionnaires.The study aims to evaluate the feasibility and acceptability of the Non Muscle Invasive Bladder Cancer-Symptom Index (NMIBC-SI)and to produce a shorter version,if appropriate
Summary of Results
The overarching aim of the research is to develop and evaluate a patient-reported Symptom Index (SI) for individuals with Non Muscle Invasive Bladder Cancer (the NMIBC-SI) that is acceptable to patients, reliable, valid and responsive to differences between contemporary treatments for NMIBC, and fit for purpose as an endpoint in clinical trials.
Background:
NMIBC is a chronic condition requiring intensive follow-up, repeated endoscopic examinations, tumour resections and intravesical treatments that can be 3-monthly and life-long. In this clinical context, patient-reported outcomes (PROs) are a critical concern for patients and their managing clinicians. PROs have enormous potential to be integral to treatment assessment and recommendations for NMIBC, however, current PRO measures are inadequate for NMIBC because they lack key NMIBC-specific symptoms and side effects associated with contemporary treatments.Methods:
We conducted a systematic review and interviewed 26 patients and 20 clinicians to develop a conceptual framework of PROs important to NMIBC. The 125 issues in the conceptual framework were phrased as questions and pre-tested in 12 cognitive interviews to develop a draft 104-item NMIBC-SI understood by and relevant to NMIBC patients. In Field Test 1, we administered the draft NMIBC-SI to patients on active treatment from nine Australian sites. Items with low prevalence, conceptual similarity or high correlations (≥0.50) were flagged for exclusion. Nine Urologists agreed the final NMIBC-SI items. In Field Test 2, patients from 16 sites across four countries completed the final version NMIBC-SI. Field test 2 was an international prospective longitudinal study involving 220 newly diagnosed NMIBC-SI patients. Clinical data and patient-completed questionnaires were collected at four time-points during treatment: before tumour resection; 1-week after resection; end of induction intravesical therapy; and 1-year follow-up. Standard psychometric tests were performed to assess the reliability, validity, responsiveness, and clinical utility of the NMIBC-SI.Key findings:
1 We have comprehensive comparative data about the short and medium-term effects of contemporary treatments for NMIBC. This information is critical for supporting patients and clinicians in informed treatment decision-making and patient-centred care, an important contribution to the management of NMIBC globally.
2 The SI is the optimum PRO measure available for use in clinics globally as a routine NMIBC outcome measure, contributing to patient-centred decision-making. We are currently preparing the NMIBC-SI validation paper for publication and dissemination.
3 Implementation of the NMIBC-SI will allow improvement in health outcomes for a significant patient population in Australia and internationally. It can:
a. improve management decisions as clinicians now have vital information to make informed evidence-based treatment decisions and include the patient-perspective, e.g. in treatment choices and dose adjustment;
b. allow individual patients in the clinic to have their PROs measured, enabling individualised decisions based on what’s important for the patient;
c. improve quality of care and QOL of patients because individual management decisions will be based on factors important to the patient, and trials and guidelines will have incorporated PROs in their analysis;
d. change policy as all NMIBC efficacy trials will be required to include the SI as an outcome measure;
e. be used to provide quality assurance at individual treatment centres.
4 The SI provides a standardised, validated, clinically relevant method of assessing treatment-related patient-outcomes so that treating clinicians are made aware of any issues troubling individual patients so they can adapt their treatment accordingly.Patient and public involvement
Planning
Consumer representative as Associate Investigator: development and format of the NMIBC-SI.
Mr. Stanford has been consulted during protocol development, development of participant consent and information sheets, ethics application, and review of the content, format and layout of the NMIBC-SI. This feedback resulted in various modifications to content of the NMIBC-SI (e.g. confusing item wording and instructions).
Execution
Consumers as research participants: development of the NMIBC-SI.
In the development process, patients treated for NMIBC participated in structured interviews to: (1) explore their understanding of the items, instructions, response options and recall period, and (2) determine whether readability was appropriate for the target population and confirm completeness of concepts covered by items in the NMIBC-SI.
Consumers as research participants: development of the NMIBC-SI.
During Field Test 1 and Field test 2, patients treated for NMIBC provided patient-reported outcome data, which was used to assess the psychometric properties of the NMIBC-SI.
Outcomes
Consumer comments and feedback directly informed modifications to the NMIBC-SI (e.g. redundant items removed, wording revised, problematic/confusing instructions modified).
Patient-reported outcome data provided by consumers, was used to assess the psychometric properties of the NMIBC-SI.Conclusions
Management of NMIBC aims to reduce progression and recurrence, thereby decreasing the incidence of bladder cancer. However, treatments cause local and systemic toxicity; e.g. BCG has proven effectiveness, but only 16% of patients complete their full treatment schedule. We used appropriate and rigorous PRO methodology to develop a new patient-reported measure designed specifically to reliably and validly assess the harms and benefits, from the patients’ perspective, of contemporary and emerging treatments for NMIBC. As an endpoint for clinical trials, the SI will influence the evolution of NMIBC treatments that are better tolerated by patients, hence better compliance and effectiveness in real world settings.REC name
London - Fulham Research Ethics Committee
REC reference
18/LO/1841
Date of REC Opinion
15 Oct 2018
REC opinion
Favourable Opinion