Erdafitinib in BCG-treated high-risk non-muscle-invasive bladder cancer
Copyright © 2023 The Author(s). Published by Elsevier Ltd.. All rights reserved..
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.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2024 |
|---|---|
| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:35 |
|---|---|
| Enthalten in: |
Annals of oncology : official journal of the European Society for Medical Oncology - 35(2024), 1 vom: 15. Jan., Seite 98-106 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Catto, J W F [VerfasserIn] |
|---|
| Links: |
|---|
| Anmerkungen: |
Date Completed 15.01.2024 Date Revised 15.01.2024 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1016/j.annonc.2023.09.3116 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM36363990X |
|---|
| LEADER | 01000caa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM36363990X | ||
| 003 | DE-627 | ||
| 005 | 20240115231902.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2024 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1016/j.annonc.2023.09.3116 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1260.xml |
| 035 | |a (DE-627)NLM36363990X | ||
| 035 | |a (NLM)37871701 | ||
| 035 | |a (PII)S0923-7534(23)04015-2 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Catto, J W F |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Erdafitinib in BCG-treated high-risk non-muscle-invasive bladder cancer |
| 264 | 1 | |c 2024 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 15.01.2024 | ||
| 500 | |a Date Revised 15.01.2024 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2023 The Author(s). Published by Elsevier Ltd.. All rights reserved. | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 520 | |a 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 | ||
| 650 | 4 | |a Randomized Controlled Trial | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a FGFR | |
| 650 | 4 | |a erdafitinib | |
| 650 | 4 | |a intravesical chemotherapy | |
| 650 | 4 | |a non-muscle-invasive bladder cancer | |
| 650 | 4 | |a recurrence-free survival | |
| 650 | 4 | |a safety | |
| 650 | 7 | |a erdafitinib |2 NLM | |
| 650 | 7 | |a 890E37NHMV |2 NLM | |
| 650 | 7 | |a BCG Vaccine |2 NLM | |
| 650 | 7 | |a Adjuvants, Immunologic |2 NLM | |
| 650 | 7 | |a Pyrazoles |2 NLM | |
| 650 | 7 | |a Quinoxalines |2 NLM | |
| 700 | 1 | |a Tran, B |e verfasserin |4 aut | |
| 700 | 1 | |a Rouprêt, M |e verfasserin |4 aut | |
| 700 | 1 | |a Gschwend, J E |e verfasserin |4 aut | |
| 700 | 1 | |a Loriot, Y |e verfasserin |4 aut | |
| 700 | 1 | |a Nishiyama, H |e verfasserin |4 aut | |
| 700 | 1 | |a Redorta, J P |e verfasserin |4 aut | |
| 700 | 1 | |a Daneshmand, S |e verfasserin |4 aut | |
| 700 | 1 | |a Hussain, S A |e verfasserin |4 aut | |
| 700 | 1 | |a Cutuli, H J |e verfasserin |4 aut | |
| 700 | 1 | |a Procopio, G |e verfasserin |4 aut | |
| 700 | 1 | |a Guadalupi, V |e verfasserin |4 aut | |
| 700 | 1 | |a Vasdev, N |e verfasserin |4 aut | |
| 700 | 1 | |a Naini, V |e verfasserin |4 aut | |
| 700 | 1 | |a Crow, L |e verfasserin |4 aut | |
| 700 | 1 | |a Triantos, S |e verfasserin |4 aut | |
| 700 | 1 | |a Baig, M |e verfasserin |4 aut | |
| 700 | 1 | |a Steinberg, G |e verfasserin |4 aut | |
| 700 | 0 | |a THOR-2 Cohort 1 Investigators |e verfasserin |4 aut | |
| 700 | 1 | |a Bengio, Ruben |e investigator |4 oth | |
| 700 | 1 | |a Cutuli, Hernan |e investigator |4 oth | |
| 700 | 1 | |a Salinas, Jorge |e investigator |4 oth | |
| 700 | 1 | |a Ameye, Filip |e investigator |4 oth | |
| 700 | 1 | |a Joniau, Steven |e investigator |4 oth | |
| 700 | 1 | |a Rodrigues da Rosa, Diogo |e investigator |4 oth | |
| 700 | 1 | |a Martins da Trindade, Karine |e investigator |4 oth | |
| 700 | 1 | |a Luz, Murilo Almeida |e investigator |4 oth | |
| 700 | 1 | |a Bavaresco, Mario Henrique |e investigator |4 oth | |
| 700 | 1 | |a de Paula, Adriano |e investigator |4 oth | |
| 700 | 1 | |a Santiag, Jose |e investigator |4 oth | |
| 700 | 1 | |a Wang, Shaogang |e investigator |4 oth | |
| 700 | 1 | |a Ye, Dingwei |e investigator |4 oth | |
| 700 | 1 | |a Boegemann, Martin |e investigator |4 oth | |
| 700 | 1 | |a Roghmann, Florian |e investigator |4 oth | |
| 700 | 1 | |a Heidrich, Albert |e investigator |4 oth | |
| 700 | 1 | |a Hellmis, Eva |e investigator |4 oth | |
| 700 | 1 | |a Faba, Óscar Rodriguez |e investigator |4 oth | |
| 700 | 1 | |a Dominguez, Jose Luis |e investigator |4 oth | |
| 700 | 1 | |a Mathieu, Romain |e investigator |4 oth | |
| 700 | 1 | |a Colombel, Marc |e investigator |4 oth | |
| 700 | 1 | |a Bladou, Franck |e investigator |4 oth | |
| 700 | 1 | |a Artignan, Xavier |e investigator |4 oth | |
| 700 | 1 | |a Vasdev, Nikhil |e investigator |4 oth | |
| 700 | 1 | |a Shimpi, Rajendra |e investigator |4 oth | |
| 700 | 1 | |a Guadalupi, Valentina |e investigator |4 oth | |
| 700 | 1 | |a Tambaro, Rosa |e investigator |4 oth | |
| 700 | 1 | |a Sirotova, Zuzana |e investigator |4 oth | |
| 700 | 1 | |a Spada, Massimiliano |e investigator |4 oth | |
| 700 | 1 | |a Necchi, Andrea |e investigator |4 oth | |
| 700 | 1 | |a Nakatsu, Hiroomi |e investigator |4 oth | |
| 700 | 1 | |a Kikuchi, Eiji |e investigator |4 oth | |
| 700 | 1 | |a Shimizu, Nobuaki |e investigator |4 oth | |
| 700 | 1 | |a Kanao, Kent |e investigator |4 oth | |
| 700 | 1 | |a Sumitomo, Makoto |e investigator |4 oth | |
| 700 | 1 | |a Naito, Yushi |e investigator |4 oth | |
| 700 | 1 | |a Ham, Won Sik |e investigator |4 oth | |
| 700 | 1 | |a Jung, Seung-Il |e investigator |4 oth | |
| 700 | 1 | |a Ha, Hongkoo |e investigator |4 oth | |
| 700 | 1 | |a Joo, Kwan Joong |e investigator |4 oth | |
| 700 | 1 | |a Ku, Ja Hyeon |e investigator |4 oth | |
| 700 | 1 | |a Seo, Ho Kyung |e investigator |4 oth | |
| 700 | 1 | |a Yun, Seokjoong |e investigator |4 oth | |
| 700 | 1 | |a Kolodziej, Anna |e investigator |4 oth | |
| 700 | 1 | |a Lawinski, Janusz |e investigator |4 oth | |
| 700 | 1 | |a Morris, David |e investigator |4 oth | |
| 700 | 1 | |a Daneshmand, Siamak |e investigator |4 oth | |
| 700 | 1 | |a Mian, Badar |e investigator |4 oth | |
| 700 | 1 | |a Lee, Eugene |e investigator |4 oth | |
| 773 | 0 | 8 | |i Enthalten in |t Annals of oncology : official journal of the European Society for Medical Oncology |d 1990 |g 35(2024), 1 vom: 15. Jan., Seite 98-106 |w (DE-627)NLM012606308 |x 1569-8041 |7 nnns |
| 773 | 1 | 8 | |g volume:35 |g year:2024 |g number:1 |g day:15 |g month:01 |g pages:98-106 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1016/j.annonc.2023.09.3116 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 35 |j 2024 |e 1 |b 15 |c 01 |h 98-106 | ||