Global real-world patients characteristics, treatment patterns, and impact of BCG shortage in patients with high-risk non-muscle invasive bladder cancer.
Authors: Jens Bedke, Anthony Eccleston, Julia Brinkmann, Jane Chang, Abin Koshy, Neil Milloy, Laure Manuel, Jacob Skilling, Emilia Biondi, Elliott Brown, Gary D. Steinberg, Oscar Rodriguez Faba, Yohann Loriot, Paolo Gontero, Yair Lotan
Journal: Journal of Clinical Oncology (2025)

717 Background: Global guidelines recommend that High Risk Non-Muscle Invasive Bladder Cancer (HR NMIBC) patients (pts) receive 1-3 years of Bacillus Calmette-Guérin (BCG) treatment (Tx). This global real-world study aimed to understand pt characteristics, Tx patterns and impact of the BCG shortage in pts with HR NMIBC. Methods: Physicians across EU5 (n=244), the US (n=55), Japan (n=44) and Canada (n=21) were recruited to the Adelphi HR NMIBC Disease Specific Programme: a cross-sectional survey with retrospective data collection extracted from medical record data for their next eight pts with HR NMIBC consecutively consulting with their physician. Tx patterns and characteristics were analysed descriptively. Data was collected from June to December 2023. Results: 364 physicians (55% urologists; 45% medical oncologists) provided data for 1930 pts with an initial diagnosis (IDx) of HR NMIBC. At IDx 73% presented with high grade T1 tumors, 21% had carcinoma in-situ, and 37% had multifocal tumors. The median time between IDx and data collection was 613 days. Tx patterns are described in the table. Of the 1395 pts who received intravesical therapy after IDx, 462 (33%) received induction Tx only, and 875 (63%) received induction followed by maintenance Tx. BCG was most commonly used induction Tx (28%), whilst 5% received intravesical chemo, similarly 56% received BCG maintenance Tx and 7% received chemo. Of those who completed BCG maintenance Tx (n=269) the median (IQR) duration was 336 (180-382) days. Physician reported BCG shortage affected 8% of HR NMIBC pts: 41% received fewer instillations, 37% received lower dosages, and 33% used different strains. Among the 29% of HR NMIBC pts who had ≥1 recurrence the median (IQR) time from IDx to 1 st recurrence was 426 (224-580) days. After 1 st recurrence, 57 pts received partial or radical cystectomies. Physicians surveyed stated that a median 20% of their overall NMIBC caseload were BCG unresponsive and 50% were BCG naïve. 45% of physicians reported having been affected by BCG shortage, ranging from 23% (Japan) to 71% (Canada), 61% reserved BCG for HR pts and 46% used intravesical chemotherapy as an alternative. Conclusions: BCG remains the most frequently prescribed Tx for HR NMIBC pts even though there have been shortage issues and many pts do not receive BCG maintenance. New Tx options that could safely reduce the amount of BCG administered without compromising pts’ outcomes are needed. Tx patterns of pts with HR NMIBC. Tx received After IDx n=1930, n (%) After 1st recurrence n=551, n (%) After 2nd recurrence n=130, n (%) TURBT + intravesical Tx 1209 (63) 194 (35) 40 (31) Other surgery + intravesical Tx 14 (1) 11 (2) 0 (0) Intravesical Tx only 172 (9) 31 (6) 10 (8) TURBT only 327 (17) 162 (29) 43 (33) Other surgery only 13 (1) 9 (2) 3 (2) Radical cystectomy only 0 (0) 44 (8) 6 (4) Other drug Tx ± surgery 88 (4) 41 (7) 9 (7) No Tx 107 (5) 59 (11) 19 (15)