Jeong Hyehyun, Jeong Jae Ho, Kim Kyu-Pyo, Lee Sang Soo, Oh Dong Wook, Park Do Hyun, Song Tae Jun, Park Yangsoon, Hong Seung-Mo, Ryoo Baek-Yeol, Yoo Changhoon
Asan Medical Center, Department of Oncology, University of Ulsan College of Medicine, Seoul 05505, Korea.
Asan Medical Center, Department of Gastroenterology, University of Ulsan College of Medicine, Seoul 05505, Korea.
Cancers (Basel). 2021 Jan 6;13(2):161. doi: 10.3390/cancers13020161.
The prognosis of advanced biliary tract cancer (BTC) is poor with the standard gemcitabine and cisplatin (GemCis) regimen. Given that the rates of human epidermal growth factor receptor 2 (HER2) positivity in BTC reaches around 15%, HER2-targeted therapy needs further investigation. This study aims to evaluate the preliminary efficacy/safety of first-line trastuzumab-pkrb plus GemCis in patients with advanced BTC. Patients with unresectable/metastatic HER2-positive BTC received trastuzumab-pkrb (on day 1 of each cycle, 8 mg/kg for the first cycle and 6 mg/kg for subsequent cycles), gemcitabine (1000 mg/m on day 1 and 8) and cisplatin (25 mg/m on day 1 and 8) every 3 weeks. Of the 41 patients screened, 7 had HER2-positive tumours and 4 were enrolled. The median age was 72.5 years (one male). Primary tumour locations included extrahepatic (N = 2) and intrahepatic (N = 1) bile ducts, and gallbladder (N = 1). Best overall response was a partial response in two patients and stable disease in two patients. Median progression-free survival (PFS) was 6.1 months and median overall survival (OS) was not reached. The most common grade 3 adverse event was neutropenia (75%), but febrile neutropenia did not occur. No patient discontinued treatment due to adverse events. Trastuzumab-pkrb with GemCis showed promising preliminary feasibility in patients with HER2-positive advanced BTC.
晚期胆管癌（BTC）采用标准的吉西他滨和顺铂（GemCis）方案治疗，预后较差。鉴于BTC中人类表皮生长因子受体2（HER2）阳性率达到15%左右，HER2靶向治疗需要进一步研究。本研究旨在评估一线曲妥珠单抗-pkrb联合GemCis治疗晚期BTC患者的初步疗效/安全性。不可切除/转移性HER2阳性BTC患者每3周接受曲妥珠单抗-pkrb（每个周期第1天，第1周期8mg/kg，后续周期6mg/kg）、吉西他滨（第1天和第8天1000mg/m²）和顺铂（第1天和第8天25mg/m²）治疗。在筛选的41例患者中，7例为HER2阳性肿瘤，4例入组。中位年龄为72.5岁（1例男性）。原发肿瘤部位包括肝外胆管（N = 2）、肝内胆管（N = 1）和胆囊（N = 1）。最佳总体反应为2例部分缓解，2例病情稳定。中位无进展生存期（PFS）为6.1个月，中位总生存期（OS）未达到。最常见的3级不良事件是中性粒细胞减少（75%），但未发生发热性中性粒细胞减少。没有患者因不良事件而停止治疗。曲妥珠单抗-pkrb联合GemCis在HER2阳性晚期BTC患者中显示出有前景的初步可行性。
