Aims
Non-muscle invasive bladder cancer (NMIBC) is a lifelong chronic condition requiring treatment and regular cystoscopies. Patient-reported outcomes (PROs) are important treatment considerations for chronic conditions; however, existing PRO measures inadequately cover NMIBC-specific symptoms and side-effects of current treatments. To address this, we developed and psychometrically evaluated a patient-reported NMIBC-Symptom Index (NMIBC-SI).
Methods
A conceptual framework of NMIBC-PROs was developed through a systematic review and interviews with patients (n=26) and clinicians (n=20). NMIBC-PROs were phrased as questions and pre-tested in cognitive interviews (n=12), producing a draft-NMIBC-SI, which was administered to 220 NMIBC-patients on active treatment. Index items were chosen based on item-level analysis for acceptability and expert consensus (n=9) on clinical relevance. The final NMIBC-SI contains a Core-index (23-items) and treatment-specific indexes: Cystoscopy (6-items) and Intravesical therapy (10-items).
A longitudinal study of newly diagnosed NMIBC patients (16 sites, 5 countries) administered the NMIBC-SI at baseline, post-surgery, post-induction, and 1-year. Composite model goodness of fit (CFI>0.9) assessed construct validity. Test-retest reliability (ICC>0.7) evaluated stability. Change from baseline, and between-group differences, assessed responsiveness and clinical validity (p<0.05).
Results
Of 232 participants (176 male; mean age 68y), 182 completed the NMIBC-SI at baseline and follow-up. Composite analysis supported a two-composite model containing the core and cystoscopy indexes (CFI=0.948). There were insufficient participant numbers to test the intravesical index. Stability reliability was supported (ICC range 0.89-0.91). Compared with baseline, Core-index symptom scores worsened post-surgery (t(401)=5.53,p<0.001), and improved post-induction (t(562)=2.89,p=0.004) and at 1-year (t(616)=3.36,p<0.001); Cyst-index showed higher symptom burden post-surgery (t(229)=7.15,p<0.001) and post-induction (t(326)=2.85,p=0.005), all supporting responsiveness. Core-index discriminated between no-treatment vs any-treatment groups (t(174)=2.89,p=0.004), and Cyst-index discriminated between surgery+chemo vs surgery alone (t(89)=2.98,p=0.004), demonstrating clinical validity.
Conclusions
The NMIBC-SI is a comprehensive and valid assessment of patient-reported NMIBC symptoms and treatment side-effects suitable for clinical research and clinical practice to improve treatment decisions and patient outcomes.