Details

Autor(en) / Beteiligte

• Narayan, Vikram M
• Boorjian, Stephen A
• Alemozaffar, Mehrdad
• Konety, Badrinath R
• Shore, Neal D
• Gomella, Leonard G
• Kamat, Ashish M
• Bivalacqua, Trinity J
• Montgomery, Jeffrey S
• Lerner, Seth P
• Busby, Joseph E
• Poch, Michael
• Crispen, Paul L
• Steinberg, Gary D
• Schuckman, Anne K
• Downs, Tracy M
• Mashni, Jr, Joseph
• Lane, Brian R
• Guzzo, Thomas J
• Bratslavsky, Gennady
• Karsh, Lawrence I
• Woods, Michael E
• Brown, Gordon
• Canter, Daniel
• Luchey, Adam
• Lotan, Yair
• Inman, Brant A
• Williams, Michael B
• Cookson, Michael S
• Chang, Sam S
• Sankin, Alexander I
• O'Donnell, Michael A
• Sawutz, David
• Philipson, Richard
• Parker, Nigel R
• Yla-Herttuala, Seppo
• Rehm, Dorte
• Jakobsen, Jørn S
• Juul, Kristian
• Dinney, Colin P N

Titel

Efficacy of Intravesical Nadofaragene Firadenovec for Patients With Bacillus Calmette-Guérin-Unresponsive Nonmuscle-Invasive Bladder Cancer: 5-Year Follow-Up From a Phase 3 Trial

Ist Teil von

• The Journal of urology, 2024-07, Vol.212 (1), p.74

Ort / Verlag

United States

Erscheinungsjahr

2024

Quelle

MEDLINE

Beschreibungen/Notizen

• Nadofaragene firadenovec-vncg is a nonreplicating adenoviral vector-based gene therapy for bacillus Calmette-Guérin (BCG)-unresponsive carcinoma in situ (CIS) with/without high-grade Ta/T1. We report outcomes following 5 years of planned follow-up. This open-label phase 3 trial (NCT02773849) enrolled patients with BCG-unresponsive nonmuscle-invasive bladder cancer in 2 cohorts: CIS ± Ta/T1 (CIS; n = 107) and Ta/T1 without CIS (Ta/T1 cohort; n = 50). Patients received 75 mL (3 × 10 vp/mL) nadofaragene firadenovec intravesically once every 3 months with cystoscopy and cytology assessments, with continued treatment offered to those remaining high grade recurrence-free (HGRF). One hundred fifty-seven patients were enrolled from 33 US sites (n = 151 included in efficacy analyses). Median follow-up was 50.8 months (interquartile range 39.1-60.0), with 27% receiving ≥ 5 instillations and 7.6% receiving treatment for ≥ 57 months. Of patients with CIS 5.8% (95% CI 2.2-12.2) were HGRF at month 57, and 15% (95% CI 6.1-27.8) of patients with high-grade Ta/T1 were HGRF at month 57. Kaplan-Meier-estimated HGRF survival at 57 months was 13% (95% CI 6.9-21.5) and 33% (95% CI 19.5-46.6) in the CIS and Ta/T1 cohorts, respectively. Cystectomy-free survival at month 60 was 49% (95% CI 40.0-57.1): 43% (95% CI 32.2-53.7) in the CIS cohort and 59% (95% CI 43.1-71.4) in the Ta/T1 cohort. Overall survival at 60 months was 80% (71.0, 86.0): 76% (64.6-84.5) and 86% (70.9-93.5) in the CIS and Ta/T1 cohorts, respectively. Only 5 patients (4 with CIS and 1 with Ta/T1) experienced clinical progression to muscle-invasive disease. At 60 months, nadofaragene firadenovec-vncg allowed bladder preservation in nearly half of the patients and proved to be a safe option for BCG-unresponsive nonmuscle-invasive bladder cancer.