Randomized trial evaluates overall survival and patient-reported outcomes in high-risk non-muscle-invasive bladder cancer, suggesting minimal impact on quality of life.
4624 Background: In the Phase 3 POTOMAC study (NCT03528694), 1 year of D in combination with BCG (I + M) resulted in a statistically significant and clinically meaningful improvement in disease-free survival vs BCG (I + M) alone, with a manageable safety profile, in patients (pts) with BCG-naive, high-risk NMIBC. We report a planned updated 5-year OS analysis and PROs. Methods: Eligible pts were randomized 1:1:1 to D + BCG (I + M), D + BCG (I only), or BCG (I + M). Secondary endpoints included 5-year OS and PROs, evaluated every 8 weeks by EORTC QLQ-C30 and every 4 weeks by EORTC QLQ-NMIBC24 and PRO-CTCAE. Prespecified priority subscales were global health status/quality of life (GHS/QoL), physical functioning, and fatigue for QLQ-C30; and urinary symptoms, intravesical treatment (tx) issues, future perspective/worries, and sexual functioning for QLQ-NMIBC24. Change from baseline (CFB; mixed model for repeated measures) was assessed; a ±10-point score was considered clinically meaningful. Results: At data cutoff Oct 3, 2025 (median follow-up 72 months), the OS HR was 0.81 (95% CI, 0.54–1.19) with a 5-year OS rate of 87.6% (95% CI, 83.5%–90.8%) for D + BCG (I + M) vs 86.3% (95% CI, 82.1%–89.6%) for BCG (I + M). Median OS was not reached in either arm. For PRO analyses (data cutoff Apr 3, 2025), QLQ-C30 and QLQ-NMIBC24 baseline compliance rates were ≥74% and ≥79%, respectively, with similar baseline scores between arms. Overall, both arms showed deterioration in QLQ-C30 adjusted mean CFB scores, with suggested clinically meaningful deterioration for fatigue with D + BCG (I + M); however, the difference between arms was small (Table). Overall QLQ-NMIBC24 adjusted mean CFB scores were numerically small and similar between arms (Table). PRO-CTCAE measures were similar between arms. Conclusions: Addition of 1 year of D to BCG (I + M) continued to show no detriment to OS at >5.5 years of follow-up and had no major impact on PROs for pts with BCG-naive high-risk NMIBC. Clinical trial information: NCT03528694 . Subscales D + BCG (I + M)Adjusted mean CFB (95% CI) a BCG (I + M)Adjusted mean CFB (95% CI) a Estimated difference of means (95% CI) QLQ-C30 GHS/QoL b −7.6 (–9.19, −6.07) −4.9 (–6.46, −3.41) −2.7 (–4.85, −0.54) Physical functioning b −5.5 (–6.76, −4.15) −2.8 (–4.11, −1.56) –2.6 (–4.43, –0.81) Fatigue c 10.1 (8.32, 11.90) 6.1 (4.37, 7.88) 4.0 (1.50, 6.46) QLQ-NMIBC24 Urinary symptoms c 1.7 (−0.06, 3.41) 0.4 (−1.27, 2.15) 1.2 (–1.19, 3.65) Intravesical tx issues c 1.5 (−0.38, 3.28) 2.7 (0.89, 4.50) −1.2 (−3.79, 1.29) Future perspective/worries c −5.5 (−7.54, −3.39) −3.2 (−5.29, −1.19) −2.2 (−5.11, 0.66) Sexual functioning b 2.2 (0.47, 3.91) 1.1 (–0.60, 2.80) 1.1 (−1.30, 3.48) a Higher scores indicate better health (GHS/functioning) or greater burden (symptoms). b Positive difference favors D + BCG (I + M). c Negative difference favors D + BCG (I + M).
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