Oncology · 4 h ago
Visugromab plus nivolumab shows activity in phase 2a bladder cancer trial
In the phase 2a GDFATHER-NEO trial, 31 patients with muscle-invasive bladder cancer received neoadjuvant nivolumab with visugromab or placebo. Pathologic complete response was observed in 33% of evaluable patients receiving visugromab and 8% receiving placebo, but the small, nonrandomized study requires confirmation.
- pCR was 33% with visugromab versus 8% with placebo.
- Trial enrolled 31 cisplatin-ineligible or cisplatin-refusing patients.
- Grade 3–4 treatment-related events occurred in 13% and 20%.
- Small, nonrandomized phase 2a findings need confirmation.
GDFATHER-NEO was an exploratory, single-blinded, nonrandomized phase 2a trial conducted at four centers in Italy. It enrolled 31 patients with newly diagnosed stage T2–T4aN0M0 muscle-invasive bladder cancer who were ineligible for or declined cisplatin. Participants were allocated sequentially within clinical-stage and PD-L1 strata to three cycles of nivolumab plus visugromab (n=16) or placebo (n=15), followed by cystectomy or, in some cases, repeat transurethral resection. Pathologic complete response (pCR) and radiographic response were co-primary endpoints.
Locally assessed pCR was 33% (5/15; 95% CI 12–62%) with visugromab and 8% (1/13; 95% CI 0–36%) with placebo. Major pathologic response was 60% versus 23%, and radiographic overall response was 60% versus 15%, respectively. Grade 3–4 treatment-related adverse events occurred in 13% and 20%; no treatment-related discontinuations or surgery delays were reported.
The findings support further study of GDF-15 blockade, but do not establish a treatment benefit: the trial was small, exploratory and nonrandomized, and confidence intervals were wide. pCR is a surrogate endpoint, and the supplied report does not provide comparative survival results. Visugromab remains investigational in this setting.
In practice
For patients with newly diagnosed muscle-invasive bladder cancer who are ineligible for or decline cisplatin, this small phase 2a study found higher observed pathologic and radiographic response rates when investigational visugromab was added to neoadjuvant nivolumab. The findings do not justify changing practice: allocation was sequential rather than randomized, the groups were small, confidence intervals were wide, and survival benefit is not established in the supplied report. Visugromab remains investigational in this setting.
AI-generated clinical interpretation · Not editor-reviewed
Is this summary clinically accurate?
Help fellow clinicians: your rating sends inaccurate summaries straight to our editors.
Sign in to rate this summary →Source
Nature Medicine: Neoadjuvant nivolumab with or without visugromab in muscle-invasive bladder cancer: the phase 2a GDFATHER-NEO trial ↗This is an automated AI-condensed summary that has not yet been reviewed by an editor. Always consult the full item at the original source.