Dr Hayne on Mitomycin/BCG as an Alternative to BCG Alone in Non–Muscle-Invasive Bladder Cancer

“In terms of safety and tolerability, [the profile of] BCG plus mitomycin was very similar to BCG alone. However, there were significantly fewer treatment discontinuations and 40% less BCG utilization in the BCG plus mitomycin arm vs the BCG alone arm.”

Dickon Hayne, MD, FRCS, MBBS, a professor of urology at The University of Western Australia Medical School, as well as a consultant urological surgeon and the head of Urology for the South Metropolitan Health Service, discussed how mitomycin plus BCG could pose a feasible alternative to BCG alone in non–muscle-invasive bladder cancer (NMIBC).

Data from the phase 3 ANZUP 1301 trial (NCT02948543), which were presented at the 2025 ASCO Annual Meeting, revealed that the intravesical combination of mitomycin and BCG demonstrated similar efficacy and safety in patients with NMIBC, Hayne began. Additionally, the combination showed an added benefit of using 40% less BCG doses than BCG alone, he noted. In the subgroup analysis, he explained that patients with high-risk NMIBC showed an increased benefit with the combination compared with BCG alone.

Of note, at a median follow-up of 48 months (interquartile range, 34-64), the disease-free survival (DFS) rate at 2 years was 75% (95% CI, 70%-81%) vs 71% (95% CI, 65%-77%) for patients treated in the mitomycin/BCG (n = 249) and BCG alone arms (n = 252), respectively (HR, 0.87; 95% CI, 0.65-1.16; P = .34). Furthermore, the complete response (CR) rate at 3 months was 90% vs 86% in the respective arms (relative risk, 1.05; 95% CI, 0.98-1.12; P = .22). In the investigational arm, 2056 doses of BCG were used compared with 3383 in the BCG monotherapy arm.

The study included patients with high-risk NMIBC with high-grade Ta or any grade T1 disease, who were eligible to receive intravesical chemotherapy. Patients were then randomly assigned to either the mitomycin/BCG arm or the BCG alone arm. In the combination arm, BCG was administered during weeks 1, 2, 4, 5, 7, and 8; mitomycin was given on weeks 3, 6, and 9 during the induction period. Conversely, those in the BCG monotherapy arm were given BCG weekly from weeks 1 to 6.

DFS served as the study’s primary end point. Other end points included CR on cystoscopy and biopsy at 3 months, time to recurrence, time to progression, overall survival, safety, and health-related quality of life.

Based on data from the trial, mitomycin plus BCG could provide a beneficial alternative to BCG alone, which is essential during the ongoing BCG shortage, Hayne emphasized. A widespread adoption of the combination could ultimately help alleviate the shortage, he concluded.