From the Division of Oncology, Department of Medicine, Mayo Clinic, Rochester, MN.
Division of Oncology, Department of Medicine, Mayo Clinic, Phoenix, AZ.
Pancreas. 2021 Apr 1;50(4):500-505. doi: 10.1097/MPA.0000000000001794.
Checkpoint inhibitors (CPIs) for low- and intermediate-grade neuroendocrine tumors (NETs) have been associated with limited efficacy; recent studies suggest CPIs may represent promising treatment for high-grade neuroendocrine neoplasms (NENs).
We examined 57 patients with NENs who were treated with CPIs to determine if NETs and neuroendocrine carcinomas (NECs) respond to immunotherapy.
Patients with poorly differentiated NECs on CPI monotherapy had an objective response rate (ORR) of 0% and median progression-free survival (PFS) of 2.1 months (95% confidence interval [CI], 0.5-4.6). Patients with poorly differentiated NECs on dual CPI therapy had an ORR of 13% and PFS of 3.5 months (95% CI, 1.4-not reached [NR]). Patients with poorly differentiated NECs on CPI and cytotoxic therapy had an ORR of 36% with PFS of 4.2 months (95% CI, 1.6-NR). Well-differentiated grade 1 and 2 NETs on CPI monotherapy had an ORR of 25% with PFS NR. Well-differentiated grade 3 NETs had 0% ORR with a PFS of 2.9 months (95% CI, 1.4-4.2) on CPI monotherapy.
Checkpoint inhibitor therapy shows limited activity in patients with NENs. Future studies should identify biomarkers that can help identify patients who are likely responders to immunotherapy.
检查点抑制剂 (CPIs) 已被证明对低级别和中级神经内分泌肿瘤 (NETs) 疗效有限；最近的研究表明 CPIs 可能是治疗高级别神经内分泌肿瘤 (NENs) 的有前途的方法。
我们研究了 57 名接受 CPIs 治疗的 NEN 患者，以确定 NETs 和神经内分泌癌 (NECs) 是否对免疫疗法有反应。
接受 CPIs 单药治疗的低分化 NEC 患者客观缓解率 (ORR) 为 0%，无进展生存期 (PFS) 中位数为 2.1 个月 (95%CI，0.5-4.6)。接受双重 CPIs 治疗的低分化 NEC 患者 ORR 为 13%，PFS 为 3.5 个月 (95%CI，1.4-NR)。接受 CPIs 和细胞毒性治疗的低分化 NEC 患者 ORR 为 36%，PFS 为 4.2 个月 (95%CI，1.6-NR)。接受 CPIs 单药治疗的高分化 1 级和 2 级 NET 患者 ORR 为 25%，PFS 无进展。接受 CPIs 单药治疗的高分化 3 级 NET 患者 ORR 为 0%，PFS 为 2.9 个月 (95%CI，1.4-4.2)。
CPIs 治疗在 NEN 患者中显示出有限的活性。未来的研究应确定有助于识别可能对免疫治疗有反应的患者的生物标志物。
