Erdafitinib in BCG-treated high-risk non-muscle-invasive bladder cancer
Catto, James (Sheffield Teaching Hospitals NHS Trust)
Tran, Ben (Peter MacCallum Cancer Centre)
Rouprêt, Morgan (Sorbonne University)
Gschwend, Jürgen (Technical University of Munich)
Loriot, Yohann (Université Paris-Saclay)
Nishiyama, Hiroyuki (University of Tsukuba)
Palou, Juan (Institut de Recerca Sant Pau)
Daneshmand, Sia (University of Southern California)
Hussain, S.A. (Sheffield Teaching Hospitals NHS Trust)
Cutuli, Hernán Javier (Sirio Libanes Hospital)
Procopio, Giuseppe (Fondazione IRCCS Istituto Nazionale dei Tumori)
Guadalupi, Valentina (Fondazione IRCCS Istituto Nazionale dei Tumori)
Vasdev, Nikhil (East and North Hertfordshire NHS Trust (Anglaterra, Regne Unit))
Naini, Vahid (Janssen Research & Development (San Diego, Estats Units d'Amèrica))
Crow, Lauren (Janssen Research & Development (Spring House, Estats Units d'Amèrica))
Triantos, Spyros (Janssen Research & Development (Spring House, Estats Units d'Amèrica))
Baig, Mahadi (Janssen Research & Development (Spring House, Estats Units d'Amèrica))
Steinberg, Gary (Rush University Medical Center (Estats Units d'Amèrica))
Bengió, Rubén
Salinas, Jorge
Ameye, Filip
Joniau, Steven
Rodrigues da Rosa, Diogo
Martins da Trindade, Karine
de Almeida Luz, Murilo
Bavaresco, Mario Henrique
de Paula, Adriano
Santiag, Jose
Wang, Shaogang
Ye, Dingwei
Boegemann, Martin
Roghmann, Florian
Heidrich, Albert
Hellmis, Eva
Rodríguez Faba, Óscar (Institut de Recerca Sant Pau)
Dominguez, Jose Luís
Mathieu, Romain
Colombel, Marc
Bladou, Franck
Artignan, Xavier
Vasdev, Nikhil
Shimpi, Rajendra
Tambaro, Rosa
Sirotova, Zuzana
Spada, Massimiliano
Necchi, Andrea
Nakatsu, Hiroomi
Kikuchi, Eiji
Shimizu, Nobuaki
Kanao, Kent
Sumitomo, Makoto
Naito, Yushi
Ham, Won Sik
Jung, Seung-Il
Ha, Hongkoo
Joo, Kwan Joong
Ku, Ja Hyeon
Seo, Ho Kyung
Yun, Seokjoong
Kolodziej, Anna
Lawinski, Janusz
Morris, David
Daneshmand, Siamak
Mian, Badar
Lee, Eugene

Date:	2024
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.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Erdafitinib ; FGFR ; Intravesical chemotherapy ; Non-muscle-invasive bladder cancer ; Recurrence-free survival ; Safety
Published in:	Annals of oncology, Vol. 35 Núm. 1 (january 2024) , p. 98-106, ISSN 1569-8041

DOI: 10.1016/j.annonc.2023.09.3116
PMID: 37871701

9 p, 400.6 KB

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