Real-world treatment patterns and clinical outcomes among US patients with non-muscle invasive bladder cancer (NMIBC) during the BCG shortage.

4612 Background: Bladder cancer is diagnosed in 80,000 US patients (pts) annually; 70% present as non-muscle invasive (NMIBC). Guidelines are risk-based; treatment for high risk (hr) disease includes tumor resection (TURBT) and intravesical therapy, commonly Bacille Calmette Guérin (BCG). Delivery of indicated treatment remains challenged, especially by ongoing (acutely since 2017) BCG shortages. We describe recent real-world (rw) patient characteristics, treatments (BCG, TURBT use), and clinical outcomes for NMIBC pts by risk group. Methods: We analyzed demographic, clinical and treatment data from electronic health records in N-Power Medicine's Real-World Analytical Dataset, largely based in the Midwest. Adults diagnosed with NIMBC between 1/2017 - 6/2022 were included and followed through 2022. Pts were classified as hr (Y/N) by AUA definition. Kaplan-Meier (KM) curves estimated BCG maintenance (MTX) duration, rw MIBC and metastatic free survival (modified PFS), cystectomy free survival (CFS), overall survival (OS), and recurrence free survival (RFS). Logistic and Cox regression was used to identify predictors of MTX start and discontinuation among hr pts including demographics, diagnosis year, smoking status, Charlson comorbidity index (CCI) and physician specialty. Results: Among 3,516 NMIBC pts (median age at diagnosis 72 years), 2,634 (75%) had hrNMIBC; most were non-Hispanic White (88%) and male (75%). Over a median follow-up of 2.7 years, 29% of hrNMIBC pts received BCG induction (IDX); 12% received BCG MTX and median MTX duration was 7.7 months (95% confidence interval [CI]: 6.8-9.2). For hr pts who received IDX, those diagnosed in 2019-2020 were less likely to initiate MTX (2019 vs 2017: OR 0.4; 95% CI [0.2-0.7], 2020 vs. 2017: OR 0.6 CI [0.4-1.0]). Hr pts with higher CCI (>2 vs 0: HR 2.2 [1.2-4.2]) were more likely to discontinue MTX. Most (95%) BCG recipients received no other therapy after BCG. Nearly all pts (>99%) received TURBT. Clinical outcomes (rwmPFS, rwCFS, rwOS) were less favorable for hr pts (Table). Conclusions: We examined treatment during the BCG shortage. BCG IDX and MTX were low among hrNMIBC pts. Clinical outcomes during the shortage highlight the potential to improve outcomes, especially for hr pts, and the need for alternative treatment strategies. KM percent survival (95% CI) of rwmPFS 1 , rwCFS 2 , and rwOS 3 by risk category. Follow up Month rwmPFS rwCFS rwOS Non-Hr Hr Non-Hr Hr Non-Hr Hr 12 97 (96, 98) 90 (89, 92) 97 (95, 98) 91 (90, 92) 90 (78, 100) 72 (69, 75) 24 92 (90, 94) 85 (83, 86) 92 (90, 94) 85 (83, 86) 85 (70, 100) 64 (60, 68) 36 89 (86, 91) 79 (77, 80) 89 (86, 91) 78 (76, 80) 85 (70, 100) 57 (53, 62) 48 87 (84, 90) 75 (73, 77) 87 (84, 90) 74 (72, 76) 75 (56, 100) 53 (48, 58) 60 81 (77, 85) 70 (68, 73) 81 (77, 85) 70 (67, 73) — (—, —) 47 (42, 53) 1 Real-world modified progression free survival. 2 Real-world cystectomy-free survival. 3 Real-world overall survival.