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Erdafitinib in BCG-treated high-risk non-muscle-invasive bladder cancer
J W F Catto1, B Tran2, M Rouprêt3
1Department of Oncology and Metabolism, University of Sheffield, Sheffield; Sheffield Teaching Hospitals NHS Trust, Sheffield, UK.
Background:
Treatment options are limited for patients with high-risk non-muscle-invasive bladder cancer (NMIBC) with disease recurrence after bacillus Calmette-Guérin (BCG) treatment and who are ineligible for/refuse radical cystectomy. FGFR alterations are commonly detected in NMIBC. We evaluated the activity of oral erdafitinib, a selective pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor, versus intravesical chemotherapy in patients with high-risk NMIBC and select FGFR3/2 alterations following recurrence after BCG treatment.
Patients And Methods:
Patients aged ≥18 years with recurrent, BCG-treated, papillary-only high-risk NMIBC (high-grade Ta/T1) and select FGFR alterations refusing or ineligible for radical cystectomy were randomized to 6 mg daily oral erdafitinib or investigator's choice of intravesical chemotherapy (mitomycin C or gemcitabine). The primary endpoint was recurrence-free survival (RFS). The key secondary endpoint was safety.
Results:
Study enrollment was discontinued due to slow accrual. Seventy-three patients were randomized 2 : 1 to erdafitinib (n = 49) and chemotherapy (n = 24). Median follow-up for RFS was 13.4 months for both groups. Median RFS was not reached for erdafitinib [95% confidence interval (CI) 16.9 months-not estimable] and was 11.6 months (95% CI 6.4-20.1 months) for chemotherapy, with an estimated hazard ratio of 0.28 (95% CI 0.1-0.6; nominal P value = 0.0008). In this population, safety results were generally consistent with known profiles for erdafitinib and chemotherapy.
Conclusions:
Erdafitinib prolonged RFS compared with intravesical chemotherapy in patients with papillary-only, high-risk NMIBC harboring FGFR alterations who had disease recurrence after BCG therapy and refused or were ineligible for radical cystectomy.
Insights
Oral erdafitinib demonstrated improved recurrence-free survival compared to intravesical chemotherapy for high-risk non-muscle-invasive bladder cancer (NMIBC) patients with FGFR alterations post-BCG treatment.
Area of Science:
- Urology
- Oncology
- Medical Research
Background:
- High-risk non-muscle-invasive bladder cancer (NMIBC) recurrence after bacillus Calmette-Guérin (BCG) treatment presents limited therapeutic options, especially for patients ineligible for or refusing radical cystectomy.
- Fibroblast growth factor receptor (FGFR) alterations are frequently observed in NMIBC, suggesting potential therapeutic targets.
Purpose of the Study:
- To evaluate the efficacy of oral erdafitinib, a selective pan-FGFR tyrosine kinase inhibitor, against intravesical chemotherapy in patients with high-risk NMIBC.
- To assess erdafitinib's activity in patients with select FGFR3/2 alterations who experienced disease recurrence after BCG treatment and were not candidates for radical cystectomy.
Main Methods:
- A randomized study comparing oral erdafitinib (6 mg daily) to investigator's choice of intravesical chemotherapy (mitomycin C or gemcitabine).
- Participants were adults with recurrent, BCG-treated, papillary-only high-risk NMIBC (high-grade Ta/T1) with select FGFR alterations, refusing or ineligible for radical cystectomy.
- The primary endpoint was recurrence-free survival (RFS); safety was a key secondary endpoint.
Main Results:
- The study was discontinued early due to slow patient accrual, with 73 patients randomized (49 to erdafitinib, 24 to chemotherapy).
- Erdafitinib showed a significantly prolonged RFS compared to chemotherapy (hazard ratio of 0.28, nominal P=0.0008). Median RFS was not reached with erdafitinib versus 11.6 months with chemotherapy.
- Safety profiles were consistent with known erdafitinib and chemotherapy data.
Conclusions:
- Erdafitinib demonstrated superior recurrence-free survival over intravesical chemotherapy in the studied NMIBC patient population.
- The findings suggest erdafitinib as a potential treatment for high-risk NMIBC with FGFR alterations after BCG failure in patients unsuitable for cystectomy.
