Roderburg Christoph, Berres Marie-Luise, Wree Alexander, Loosen Sven H, Luedde Tom, Trautwein Christian
Department of Medicine III, University Hospital RWTH Aachen, Aachen, Germany.
Division of Gastroenterology, Hepatology and Hepatobiliary Oncology, Aachen, Germany.
Visc Med. 2019 Mar;35(1):43-46. doi: 10.1159/000497465. Epub 2019 Feb 12.
Hepatocellular carcinoma (HCC) represents the most common primary carcinoma of the liver. Most patients present with advanced or metastatic HCC at diagnosis and face a very limited prognosis. Systemic treatment with the tyrosine-kinase inhibitors sorafenib or lenvatinib is considered as the treatment of choice in these patients. In patients intolerant to tyrosine-kinase inhibitors, no standard therapy is approved so far, but several agents have demonstrated efficacy in clinical trials. As such, the checkpoint inhibitors nivolumab and pembrolizumab have been shown to induce tumor response and to prolong survival in patients with advanced HCC, both when intolerant to sorafenib or after failure of a first-line therapy with sorafenib.
We here report the case of a patient with advanced HCC that demonstrated severe toxicity upon first-line treatment with sorafenib. Administration of sorafenib was discontinued and after careful consideration the patient started a second-line treatment with the PD-1 antibody nivolumab. Strikingly, this treatment led to a significant regression of tumor size and a drastic reduction of alpha-fetoprotein serum concentrations. At 17 months after initiation of treatment, the patient is still alive in excellent condition with sustained tumor response.
In summary, we report on a very rare case of a patient with HCC demonstrating an almost complete response to checkpoint inhibitor treatment.
肝细胞癌（HCC）是最常见的原发性肝癌。大多数患者在诊断时已出现晚期或转移性HCC，预后非常有限。酪氨酸激酶抑制剂索拉非尼或仑伐替尼的全身治疗被认为是这些患者的首选治疗方法。对于不耐受酪氨酸激酶抑制剂的患者，目前尚无标准疗法获批，但有几种药物在临床试验中已显示出疗效。因此，检查点抑制剂纳武单抗和派姆单抗已被证明在晚期HCC患者中可诱导肿瘤反应并延长生存期，无论是对索拉非尼不耐受的患者还是一线索拉非尼治疗失败后的患者。
我们在此报告一例晚期HCC患者，该患者在一线使用索拉非尼治疗时出现严重毒性。停用索拉非尼后，经过仔细考虑，患者开始使用PD-1抗体纳武单抗进行二线治疗。令人惊讶的是，这种治疗导致肿瘤大小显著缩小，甲胎蛋白血清浓度大幅降低。在开始治疗17个月后，患者仍然存活，状况良好，肿瘤反应持续。
总之，我们报告了一例非常罕见的HCC患者，该患者对检查点抑制剂治疗表现出几乎完全的反应。
