Department of Medicine, Hospital of the University of Pennsylvania, Philadelphia.
Department of Radiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia.
JAMA Netw Open. 2021 Mar 1;4(3):e212274. doi: 10.1001/jamanetworkopen.2021.2274.
Peptide receptor radionuclide therapy (PRRT) is approved in the US for treatment of gastroenteropancreatic neuroendocrine tumors (NETs), but data on PRRT outcomes within US populations remain scarce.
To analyze the first 2 years of PRRT implementation at a US-based NET referral center.
DESIGN, SETTING, AND PARTICIPANTS: This cohort study was conducted using medical records of patients with metastatic NET receiving PRRT from 2018 through 2020 in a NET program at a tertiary referral center. Included patients were those at the center with metastatic NETs who received at least 1 dose of PRRT over the study period. Laboratory toxic effects were assessed using Common Terminology Criteria for Adverse Events version 5.0. Tumor response was determined using Response Evaluation Criteria in Solid Tumors 1.1. Survival analysis was conducted to identify factors associated with progression-free survival (PFS) and overall survival. Data were analyzed from August 2018 through August 2020.
Receiving 4 cycles of lutetium-177-dotatate infusion, separated by 8-week intervals targeted to 7.4 GBq (200 mCi) per dose.
Data were compared from before and after PRRT to determine hematologic, liver, and kidney toxic effects and to assess tumor progression and patient survival.
Among 78 patients receiving at least 1 dose of PRRT, median (interquartile range) age at PRRT initiation was 59.8 (53.5-69.2) years and 39 (50.0%) were men. The most common primary NET sites included small bowel, occurring in 34 patients (43.6%), and pancreas, occurring in 22 patients (28.2%). World Health Organization grade 1 or 2 tumors occurred in 62 patients (79.5%). Among all patients, 56 patients underwent pretreatment with tumor resection (71.8%), 49 patients received nonsomatostatin analogue systemic therapy (62.8%), and 49 patients received liver-directed therapy (62.8%). At least 1 grade 2 or greater toxic effect was found in 47 patients (60.3%). Median PFS was 21.6 months for the study group, was not reached by 22 months for patients with small bowel primary tumors, and was 13.3 months for patients with pancreatic primary tumors. Having a small bowel primary tumor was associated with a lower rate of progression compared with having a pancreatic primary tumor (hazard ratio, 0.19; 95% CI, 0.07-0.55; P = .01). Median overall survival was not reached.
This cohort study of patients with metastatic NETs found that PRRT was associated with laboratory-measured toxic effects during treatment for most patients and an overall median PFS of 21.6 months. Patients with small bowel NETs had longer PFS after PRRT compared with patients with pancreatic NETs.
肽受体放射性核素疗法（PRRT）已获美国批准用于治疗胃肠胰神经内分泌肿瘤（NETs），但美国人群中关于 PRRT 结果的数据仍然很少。
分析美国一家 NET 转诊中心实施 PRRT 的头 2 年情况。
设计、地点和参与者：本队列研究使用了一家三级转诊中心 NET 项目中 2018 年至 2020 年期间接受 PRRT 的转移性 NET 患者的病历。纳入标准为中心内患有转移性 NET 并在研究期间至少接受过 1 剂 PRRT 的患者。使用不良事件通用术语标准 5.0 评估实验室毒性效应。使用实体瘤反应评估标准 1.1 确定肿瘤反应。生存分析用于确定与无进展生存期（PFS）和总生存期相关的因素。数据于 2018 年 8 月至 2020 年 8 月进行分析。
接受 4 个周期的镥-177- dotatate 输注，每 8 周为一个周期，每个周期的目标剂量为 7.4GBq（200mCi）。
比较 PRRT 前后的数据，以确定血液学、肝脏和肾脏毒性效应，并评估肿瘤进展和患者生存情况。
在至少接受过 1 剂 PRRT 的 78 名患者中，PRRT 开始时的中位（四分位间距）年龄为 59.8（53.5-69.2）岁，39 名（50.0%）为男性。最常见的原发 NET 部位包括小肠，发生于 34 例患者（43.6%），胰腺发生于 22 例患者（28.2%）。发生 1 级或 2 级肿瘤的患者有 62 例（79.5%）。所有患者中，56 例行肿瘤切除术预处理（71.8%），49 例接受非生长抑素类似物全身治疗（62.8%），49 例接受肝脏定向治疗（62.8%）。至少有 1 例 2 级或更高级别的毒性反应发生在 47 例患者（60.3%）中。研究组的中位 PFS 为 21.6 个月，22 个月时小肠原发肿瘤患者未达到，胰腺原发肿瘤患者的 PFS 为 13.3 个月。与胰腺原发肿瘤相比，小肠原发肿瘤患者的进展率较低（风险比，0.19；95%CI，0.07-0.55；P=0.01）。中位总生存期未达到。
这项针对转移性 NET 患者的队列研究发现，PRRT 与大多数患者在治疗期间的实验室测量毒性效应相关，总体中位 PFS 为 21.6 个月。与胰腺 NET 患者相比，接受 PRRT 的小肠 NET 患者的 PFS 更长。
