Non-muscle invasive bladder cancer (NMIBC) is a chronic condition requiring frequent follow-up with endoscopic examinations, tumour resections and intravesical treatments. In this clinical context, patient-reported outcomes (PROs) have enormous potential to inform treatment assessment and recommendations for NMIBC. We aimed to develop and evaluate a patient-reported NMIBC Symptom Index (NMIBC-SI) to facilitate clinical research and enhance care.
NMIBC-SI items were developed based on existing literature and qualitative interviews with patients and clinicians, and evaluated in two field tests: item reduction, using NMIBC-SI data from 220 patients on active treatment from nine Australian centres; reliability and validity evaluation of item-reduced version using NMIBC-SI data from 232 patients from five countries.
NMIBC-SI assesses disease and treatment-related symptom burden and two treatment-specific side-effects (cystoscopy, intravesical BCG/Chemotherapy). Composite analysis supported a single composite model including core symptom and cystoscopy index items (Intravesical index items were not tested due to small sample). Test-retest reliability was strong (range 0.894-0.91). As expected, the NMIBC-SI was able to discriminate between no treatment and any treatment groups, and no treatment and chemo/BCG groups, providing evidence towards validity.
NMIBC-SI assesses patients' self-reported symptom burden and can be used to evaluate NMIBC treatments from the perspective of patients. The NMIBC-SI is acceptable to patients and has evidence for reliability and validity. Future validation work with patients with greater symptom burden is warranted.
Patients with NMIBC require long-term monitoring with regular endoscopic examinations and various treatments. This research describes the development of the NMIBC-SI, a patient-completed questionnaire that assesses the impact of those treatments on health outcomes important to patients. The NMIBC-SI can be used in research and in the clinic to improve communication and treatment decisions.
Journal of patient-reported outcomes. 2025 Mar 27*** epublish ***
Claudia Rutherford, Margaret-Ann Tait, Daniel S J Costa, Madeleine T King, David P Smith, Shomik Sengupta, Joseph Ischia, Andrew Mitterdorfer, Dickon Hayne, Roger Watson, Paul Anderson, Mark Frydenberg, Peter Gilling, Nicholas Buchan, Euan Green, Noel Clarke, Stephen A Boorjian, Badrinath Konety, Jeffrey M Holzbeierlein, Peter C Black, Venu Chalasani, Jörg Henseler, Manish I Patel, NMIBC-SI Study Group
Faculty of Medicine and Health, Susan Wakil School of Nursing and Midwifery, University of Sydney, Sydney, Australia. ., Faculty of Medicine and Health, Susan Wakil School of Nursing and Midwifery, University of Sydney, Sydney, Australia., Faculty of Science, School of Psychology, University of Sydney, Sydney, NSW, Australia., The Daffodil Centre, The University of Sydney, A Joint Venture with Cancer Council New South Wales, Sydney, NSW, Australia., Eastern Health Clinical School, Monash University, Melbourne, VIC, Australia., Department of Surgery, University of Melbourne, Austin Health, Heidelberg, VIC, Australia., Concord Repatriation General Hospital, Concord, NSW, Australia., ANZUP Cancer Trials Group, Sydney, NSW, Australia., Monash Medical Centre, Department of Surgery, Monash University, Melbourne, VIC, Australia., Tauranga Urology Research, Tauranga, New Zealand., The Christie and Salford Royal Hospitals, Manchester, UK., Mayo Clinic, Rochester, MN, USA., University of Minnesota, Minneapolis, MN, USA., University of Kansas Health System, Kansas City, KS, USA., University of British Columbia, Vancouver, BC, Canada., University of Twente, Enschede, The Netherlands.