Pembrolizumab With Anlotinib in Advanced Oral Mucosal Melanoma: A Retrospective Trial

INTRODUCTION: Oral mucosal melanoma (OMM) is a rare subtype of melanoma but exhibits highly invasive biological behavior. Programmed cell death protein 1 (PD-1) monotherapy showed lower response in OMM than other subtypes of melanoma. MATERIALS AND METHODS: We retrospectively analyzed the efficacy and safety of pembrolizumab with anlotinib in patients with advanced OMM between August 2018 and September 2024. The primary endpoint was objective response rate (ORR); the secondary endpoints included disease control rate (DCR), duration of response (DOR), progression-free survival (PFS), overall survival (OS), and treatment-related adverse events (TRAEs). RESULTS: Forty-one patients were enrolled in our study. Seventeen patients (41.4%) achieved an objective response. The median PFS was 6.4 months (95% confidence interval [CI], 4.5-8.3 months), and the median OS was 10.0 months (95% CI, 8.3-11.7 months). Thirty-three patients (80.5%) experienced at least one TRAE. The most common TRAEs were hypertension (29.3%), hand-foot syndrome (19.5%), and anemia (19.5%). Grade 3 or higher TRAEs occurred in two patients (4.8%), and no grade 5 TRAEs were observed. CONCLUSIONS: Our study suggested that pembrolizumab with anlotinib in patients with advanced OMM showed potential efficacy and manageable adverse effects. Further studies are needed to confirm the efficacy of this combination strategy.