在合并型肝细胞-胆管细胞癌（cHCC-CCA）患者中，目前还没有就最佳的全身治疗方案达成一致的共识。我们描述了cHCC-CCA患者的临床特征和预后情况，特别关注接受姑息性全身治疗，包括免疫检查点抑制剂（ICIs）的患者。在这项欧洲回顾性、多中心研究中，纳入了2003年4月至2022年6月在四家机构接受组织学证实的cHCC-CCA治疗的患者。在接受姑息性全身治疗的患者中，比较了化疗和非细胞毒性药物（nCHT）治疗的患者的预后情况。在101名患者中，大多数为男性（n = 70，69 %），平均年龄为64.6 ± 10.6岁。只有一线治疗类型与总生存期（OS）有独立相关性。共对44（44%）名患者进行姑息性全身治疗。其中，25（57%）名患者接受了化疗，19（43%）名患者接受了非细胞毒性药物（n = 其中16名接受了索拉非尼）作为全身一线治疗。虽然整体反应率（ORR；化疗与nCHT：8%与5%），疾病控制率（24%与21%）和中位无进展生存期{3.0个月[95%置信区间（CI）1.4-4.6个月]与3.2个月（95% CI 2.8-3.6个月），P = 0.725}没有显著差异，但化疗组的中位OS倾向于较长[15.5个月（95% CI 8.0-23.0个月）与5.3个月（95% CI 0-12.5个月），P = 0.052]。但是，在多变量分析中，一线疗法类型（化疗与索拉非尼）与OS无关。接受ICIs药物治疗的患者（n = 7）的ORR为29%。在cHCC-CCA患者中，接受化疗和接受非细胞毒性药物的患者之间的OS、无进展生存期、ORR和疾病控制率没有显著差异。一些患者可能对免疫治疗有效。需要进行前瞻性研究以确定cHCC-CCA的最佳全身治疗方案。版权所有©2023作者。由Elsevier Ltd.出版。保留所有权利。

There is no clear consensus on the optimal systemic treatment regimen in combined hepatocellular-cholangiocarcinoma (cHCC-CCA) patients. We describe clinical characteristics and outcome of cHCC-CCA patients, with a special focus on patients receiving palliative systemic therapy, including immune checkpoint inhibitors (ICIs).In this European retrospective, multicenter study, patients with histologically proven cHCC-CCA treated at four institutions between April 2003 and June 2022 were included. In patients receiving palliative systemic therapy, outcome was compared between cytotoxic chemotherapy (CHT)- and non-cytotoxic CHT (nCHT)-treated patients.Of 101 patients, the majority were male (n = 70, 69%) with a mean age of 64.6 ± 10.6 years. Only type of first-line treatment was independently associated with overall survival (OS). Palliative systemic therapy was administered to 44 (44%) patients. Of those, 25 (57%) patients received CHT and 19 (43%) had nCHT (n = 16 of them sorafenib) in systemic first line. Although there was no significant difference in overall response rate (ORR; CHT versus nCHT: 8% versus 5%), disease control rate (24% versus 21%), and median progression-free survival {3.0 months [95% confidence interval (CI) 1.4-4.6 months] versus 3.2 months (95% CI 2.8-3.6 months), P = 0.725}, there was a trend towards longer median OS in the CHT group [15.5 months (95% CI 8.0-23.0 months) versus 5.3 months (95% CI 0-12.5 months), P = 0.052]. However, in multivariable analysis, type of first-line regimen (CHT versus sorafenib) was not associated with OS. ORR in patients receiving ICIs (n = 7) was 29%.In patients with cHCC-CCA, OS, progression-free survival, ORR, and disease control rate were not significantly different between individuals receiving CHT and patients receiving nCHT. Immunotherapy may be effective in a subset of patients. Prospective studies are needed to identify optimal systemic treatment regimens in cHCC-CCA.Copyright © 2023 The Author(s). Published by Elsevier Ltd.. All rights reserved.