- Design
- Phase I dose-escalation trial, single arm
- Population
- 25 patients with BCG-exposed high-grade non-muscle-invasive bladder cancer
- Primary outcome
- Maximally tolerated dose and safety of gemcitabine plus BCG
- Effect
- No grade 3 or higher treatment-related events; complete response 24 of 25 (96%) at 6 months
A phase I dose-escalation trial treated 25 patients with BCG-exposed high-grade non-muscle-invasive bladder cancer, 68% with carcinoma in situ, using intravesical gemcitabine every three weeks for eight doses, escalated from 500 to 2000 mg, alternating with weekly BCG for six doses.
No treatment-related grade 3 or higher adverse events or dose-limiting toxicities occurred; 72% reported grade 1 and 20% grade 2 events. At 6 months 24 of 25 (96%, 95% CI 80% to 100%) had a complete clinical response. Urinary T-cell recruiting chemokines rose and IL-6 fell.
This is a single-arm phase I study designed to find a tolerable dose, with a small sample and short follow-up. A randomised phase III comparing it with BCG retreatment is under way. Response rates in single-arm studies tend to fall in larger and longer series, so it is a reason to look out for that trial, not to change practice.
- Treat as early evidence from 25 patients with no comparison arm.
- Complete response at 6 months was 24 of 25; durability is not yet known.
- No grade 3 or higher treatment-related adverse events were recorded.
- Watch for the randomised phase III before using the combination outside a trial.
Why it matters
It offers a rational way to improve BCG retreatment, where about half of patients recur.
Don't overread it
Phase I, single-arm, 25 patients; efficacy was a secondary outcome.
The statistics, in plain English
A 96% response with a 95% interval of 80% to 100% shows how wide the uncertainty is with 25 patients. Complete response at 6 months does not tell you how many remain free of recurrence later.
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