Multinational phase 3 trial of nivolumab-ipilimumab followed by nivolumab for advanced melanoma in diverse patient groups

Previous studies demonstrated clinical benefit with immunotherapy in patients with advanced melanoma, but there is a paucity of data in patient groups with poor prognoses who are typically excluded from clinical trials (ocular/uveal melanoma, brain metastases, ECOG PS ≥ 2). The multinational phase 3b CheckMate 401 trial evaluated first-line four cycles of nivolumab-ipilimumab followed by nivolumab for ≤ 24 months for unresectable stage III of IV melanoma. The treatment was tolerable in patients with poor prognostic characteristics. Efficacy was similar between the all-treated population and patients with brain metastases. Reduced efficacy was observed in patients with ECOG PS 2, ocular/uveal melanoma, and/or mucosal melanoma. The authors conclude that there is a continued need for novel treatments for these difficult-to-treat patients.