粘膜黑色素瘤是一种罕见的黑色素瘤亚型，约占所有诊断黑色素瘤的 1%。其特点是侵袭性表型，预后不良，对已批准治疗的反应率低。我们回顾性分析了 2018 年至 2021 年间在美国一家转诊中心接受阿西替尼 ± 抗程序性细胞死亡蛋白 1 (PD-1) 治疗的粘膜黑色素瘤患者的临床特征、治疗和结果。放射学反应根据实体瘤疗效评估标准 (RECIST)，v1.1。本研究包括二十三名患者。总体而言，78% 是女性，中位年龄为 62 岁。粘膜黑色素瘤的起源部位是鼻窦（35%）、泌尿生殖道（35%）和胃肠道（30%）。 65% 的患者患有 M1c 或 M1d 疾病，0% 的患者检测到 BRAF V600 突变。大多数 (96%) 患者既往接受过包括抗 PD-1 在内的治疗，中位治疗时间为 2 次，78% 的患者接受了阿西替尼和 PD-1 联合治疗，中位治疗持续时间为 3.2 个月。总体缓解率为13%，疾病控制率为26%。中位无进展生存期为 3.2 个月，中位总生存期为 8.2 个月。总体而言，该方案耐受性良好，39% 的患者需要减少剂量，9% 的患者需要停止治疗。阿西替尼联合抗 PD-1 疗法在亚洲以外接受过多次治疗的粘膜黑色素瘤患者中具有适度的临床活性，其中一些患者具有长期获益。该数据支持评估该组合的全球临床试验以及将基于血管内皮生长因子的疗法纳入粘膜黑色素瘤患者治疗模式中的作用。版权所有 © 2024 Wolters Kluwer Health, Inc. 保留所有权利。

Mucosal melanoma is a rare melanoma subtype, accounting for about 1% of all diagnosed melanomas. It is characterized by an aggressive phenotype with a poor prognosis and a low response rate to approved treatments. We retrospectively analyzed the clinical features, treatments, and outcomes of patients diagnosed with mucosal melanoma treated with axitinib ± anti-programmed cell death protein 1 (PD-1) therapy at a single US referral center between 2018 and 2021. Radiologic response was assessed according to Response Evaluation Criteria in Solid Tumors (RECIST), v1.1. Twenty-three patients were included in this study. In all, 78% were females with a median age of 62 years. The originating site of mucosal melanoma was the sinonasal (35%), genitourinary (35%), and gastrointestinal (30%) tracts. Sixty-five percent of patients had M1c or M1d disease and 0% had BRAF V600 mutations detected. The majority (96%) had prior treatment inclusive of anti-PD-1, with a median of 2 prior lines, and 78% of patients received a combination of axitinib and PD-1 and the median duration of treatment was 3.2 months. The overall response rate was 13% and the disease control rate was 26%. The median progression-free survival was 3.2 months, and the median overall survival was 8.2 months. Overall, the regimen was well tolerated with 39% of patients requiring dose reduction and 9% requiring treatment cessation. Axitinib with anti-PD-1 therapy has modest clinical activity in heavily pretreated patients with mucosal melanoma outside of Asia, including some with long-term benefits. This data supports the worldwide clinical trials evaluating this combination and the role of incorporating vascular endothelial growth factor-based therapy in the therapeutic paradigm for patients with mucosal melanoma.Copyright © 2024 Wolters Kluwer Health, Inc. All rights reserved.