| Title: |
Erdafitinib in BCG-treated high-risk non-muscle-invasive bladder cancer |
| Authors: |
Catto, J.W.F.; Tran, B.; Rouprêt, M.; Gschwend, J.E.; Loriot, Y.; Nishiyama, H.; Redorta, J.P.; Daneshmand, S.; Hussain, S.A.; Cutuli, H.J.; Procopio, G.; Guadalupi, V.; Vasdev, N.; Naini, V.; Crow, L.; Triantos, S.; Baig, M.; Steinberg, G.; Bengio, R.; Cutuli, H.; Salinas, J.; Ameye, F.; Joniau, S.; Rodrigues da Rosa, D.; Martins da Trindade, K.; Luz, M.A.; Bavaresco, M.H.; de Paula, A.; Santiag, J.; Wang, S.; Ye, D.; Boegemann, M.; Roghmann, F.; Heidrich, A.; Hellmis, E.; Faba, ÓR.; Dominguez, J.L.; Mathieu, R.; Colombel, M.; Bladou, F.; Artignan, X.; Shimpi, R.; Tambaro, R.; Sirotova, Z.; Spada, M.; Necchi, A.; Nakatsu, H.; Kikuchi, E.; Shimizu, N.; Kanao, K.; Sumitomo, M.; Naito, Y.; Ham, W.S.; Jung, S.-I.; Ha, H.; Joo, K.J.; Ku, J.H.; Seo, H.K.; Yun, S.; Kolodziej, A.; Lawinski, J.; Morris, D.; Mian, B.; Lee, E. |
| Publisher Information: |
Elsevier BV |
| Publication Year: |
2024 |
| Collection: |
White Rose Research Online (Universities of Leeds, Sheffield & York) |
| Description: |
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. |
| Document Type: |
article in journal/newspaper |
| File Description: |
text |
| Language: |
English |
| ISSN: |
0923-7534 |
| Relation: |
https://eprints.whiterose.ac.uk/id/eprint/232772/1/PIIS0923753423040152.pdf; Catto, J.W.F. orcid.org/0000-0003-2787-8828 , Tran, B., Rouprêt, M. et al. (64 more authors) (2024) Erdafitinib in BCG-treated high-risk non-muscle-invasive bladder cancer. Annals of Oncology, 35 (1). pp. 98-106. ISSN: 0923-7534 |
| Availability: |
https://eprints.whiterose.ac.uk/id/eprint/232772/; https://eprints.whiterose.ac.uk/id/eprint/232772/1/PIIS0923753423040152.pdf |
| Rights: |
cc_by_4 |
| Accession Number: |
edsbas.EB5FB377 |
| Database: |
BASE |