Phase II trial reveals meaningful antitumor activity with first-line axitinib plus nivolumab in advanced mucosal melanoma, suggesting VEGF and PD-1 blockade overcomes immune resistance.
Key Points
To evaluate the efficacy and safety of first-line nivolumab combined with axitinib in advanced mucosal melanoma and explore treatment escalation upon disease progression.
Single-center, investigator-initiated phase II trial enrolling patients with previously untreated unresectable or metastatic mucosal melanoma (N=20 evaluable).
Patients received nivolumab every 4 weeks combined with axitinib twice daily, with a sequential phase Ib cohort testing escalation to ipilimumab or stereotactic body radiotherapy upon progression.
The confirmed objective response rate was 45% (95% CI, 23–68), including 4 complete responses (20%) and 5 partial responses (25%), with a median duration of response of 21 months (95% CI, 8.6–NR).
Median progression-free survival was 6.4 months (95% CI, 3.5–NR), and median overall survival was not reached.
Grade 3–5 treatment-related adverse events occurred in 67% of patients, including two treatment-related deaths; no unexpected toxicities occurred among the 7 patients receiving post-progression triplet therapy.