Erdafitinib in BCG-treated high-risk non-muscle-invasive bladder cancer

Catto, JWF; Tran, B; Roupr&#234;t, M; Gschwend, JE; Loriot, Y; Nishiyama, H; Redorta, JP; Daneshmand, S; Hussain, SA; Cutuli, HJ; Procopio, G; Guadalupi, V; Vasdev, N; Naini, V; Crow, L; Triantos, S; Baig, M; Steinberg, G; THOR-2 Cohort 1 Investigators

Author(s): Catto, JWF; Tran, B; Rouprêt, M; Gschwend, JE; Loriot, Y; Nishiyama, H; Redorta, JP; Daneshmand, S; Hussain, SA; Cutuli, HJ; Procopio, G; Guadalupi, V; Vasdev, N; Naini, V; Crow, L; Triantos, S; Baig, M; Steinberg, G; THOR-2 Cohort 1 Investigators;
Details: Publication Year 2024-01,Volume 35,Issue #1,Page 98-106
Journal Title: Annals of Oncology
Publication Type: Research article
Abstract: 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.
Publisher: Elsevier
Keywords: Humans; Adolescent; Adult; BCG Vaccine/adverse effects; *Non-Muscle Invasive Bladder Neoplasms; Adjuvants, Immunologic/therapeutic use; Neoplasm Recurrence, Local/drug therapy; *Urinary Bladder Neoplasms; Neoplasm Invasiveness; *Pyrazoles; *Quinoxalines; Fgfr; erdafitinib; intravesical chemotherapy; non-muscle-invasive bladder cancer; recurrence-free survival; safety
Department(s): Medical Oncology
Publisher's Version: https://doi.org/10.1016/j.annonc.2023.09.3116
Open Access at Publisher's Site: https://doi.org/10.1016/j.annonc.2023.09.3116
Terms of Use/Rights Notice: Refer to copyright notice on published article.

Creation Date: 2024-01-30 06:27:04

Last Modified: 2024-01-30 06:27:34