Department of Internal Medicine, Division of Medical Oncology, The Ohio State University Wexner Medical Center, The James Comprehensive Cancer Center, Columbus, Ohio, USA.
Department of Surgery, Division of Surgical Oncology, The Ohio State University Wexner Medical Center, The James Comprehensive Cancer Center, Columbus, Ohio, USA.
Expert Opin Investig Drugs. 2019 Nov;28(11):941-949. doi: 10.1080/13543784.2019.1677606. Epub 2019 Oct 12.
: Hepatocellular carcinoma (HCC) is the most common primary liver cancer and the burden of disease is increasing globally. Until recently, systemic therapies for HCC were limited and prognosis for advanced disease generally poor.: This article describes some recent phase I and II clinical trials for the treatment of HCC. We performed a search on Pubmed with keywords hepatocellular carcinoma, phase I clinical trial, phase II clinical trial, and immunotherapy. We also searched https://clinicaltrials.gov and identified relevant trials listed as active. Studies in progress or recently reported were conducted using novel therapies based on targets identified through molecular profiling of tumors or based on insights into immune system dysregulation in HCC. We also identified studies using drugs targeting recently discovered biomarkers such as endoglin or aldo-keto reductase 1c3. The major outcomes were safety and efficacy as measured by response rate, progression-free survival or overall survival.: HCC is a heterogeneous disease resulting from aberrations in intracellular signaling and immune system dysregulation. Thus, a multisystem approach will be required to deliver personalized therapy. Combination therapies are likely to be future options; it is also possible that modulation of the microbiome might form part of future treatment paradigms.
肝细胞癌（HCC）是最常见的原发性肝癌，疾病负担在全球范围内不断增加。直到最近，HCC 的系统治疗方法还很有限，晚期疾病的预后通常较差。本文介绍了一些最近针对 HCC 治疗的 I 期和 II 期临床试验。我们在 Pubmed 上使用了肝细胞癌、I 期临床试验、II 期临床试验和免疫疗法等关键词进行了搜索。我们还在 https://clinicaltrials.gov 上进行了搜索，并确定了列为活跃状态的相关试验。正在进行或最近报告的研究使用了基于肿瘤分子谱识别的靶点或基于 HCC 中免疫系统失调的见解的新型疗法。我们还确定了使用靶向最近发现的生物标志物（如内胚层或醛酮还原酶 1c3）的药物的研究。主要结果是通过反应率、无进展生存期或总生存期来衡量的安全性和疗效。HCC 是一种异质性疾病，是由于细胞内信号转导和免疫系统失调引起的。因此，需要采用多系统方法来提供个性化治疗。联合治疗可能是未来的选择；调节微生物组也可能成为未来治疗模式的一部分。
