Liver Cancer最新文献

{"title":"Adjuvant Atezolizumab-Bevacizumab after Resection or Ablation for Hepatocellular Carcinoma.","authors":"Masatoshi Kudo","doi":"10.1159/000531225","DOIUrl":"https://doi.org/10.1159/000531225","url":null,"abstract":"Hepatic resection and radiofrequency ablation (RFA)/ microwave ablation are well-established curative treatments for hepatocellular carcinoma (HCC); however, the disease often recurs [1, 2]. Pathological studies of resected HCC specimens reported that microscopic intrahepatic metastases are present in about 10% of patients with a solitary HCC measuring 2 cm or less. Microvascular invasion is also present in about 27% of patients [3]. Based on these facts, it is believed that there is a certain risk of recurrence of intrahepatic metastasis, even for a single nodule measuring 2 cm or less. Furthermore, the larger the tumor, or the presence of multiple HCCs larger than 2 cm, increases the risk of intrahepatic metastasis and microvascular invasion, and therefore the risk of intrahepatic metastatic recurrence [4, 5]. There are two patterns of recurrence after curative treatment: early and late. Early recurrence involves mainly intrahepatic metastasis via the portal vein, while late recurrence has a more multicentric etiology [2]. About 80% of HCCs recur 5 years after RFA or resection [6]. The main reason for the poor prognosis associated with HCC is frequent recurrence, even after curative treatment. Repeated TACE to treat recurrence worsens liver function in many cases, resulting in death from liver failure. Conversely, if HCC recurrence after curative treatment can be suppressed, the prognosis should improve dramatically. Several adjuvant trials have been conducted to inhibit recurrence, but all yielded negative results [7–9]. To date, representative clinical trials of adjuvants that help to prevent recurrence include a trial of Vitamin K [7], the NIK-333 trial that used retinoid [8], and the STORM trial","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 3","pages":"189-197"},"PeriodicalIF":13.8,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/f4/57/lic-2023-0012-0003-531225.PMC10360452.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10291505","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Genomic and Immune Features in an Intrahepatic Cholangiocarcinoma Patient with Microsatellite Instability-High Suffered Rapid Acquired Resistance to PD-1 Inhibitor.","authors":"Zhuo Cheng,&nbsp;Tianmei Zeng,&nbsp;Guang Yang,&nbsp;Di Liu,&nbsp;Zhi Zheng,&nbsp;Zhengang Yuan","doi":"10.1159/000530273","DOIUrl":"https://doi.org/10.1159/000530273","url":null,"abstract":"<p><strong>Introduction: </strong>Intrahepatic cholangiocarcinoma (ICC) is a highly aggressive liver malignancy with poor prognosis. Recently, the development of immune checkpoint inhibitors (ICIs), such as programmed cell death 1 (PD-1) inhibitors, has emerged as a promising strategy in multiple tumor types, including ICC. Microsatellite instability-high (MSI-H) is an important biomarker for ICIs in solid tumors. The response rate in patients with MSI-H is significantly higher than in those with microsatellite stability/microsatellite instability-low. And approximately 80-90% of the patients with MSI-H could maintain sustained clinical benefits once they had an initial response. However, some patients could have primary resistance at the beginning, and some might have acquired resistance after long-term treatment.</p><p><strong>Case presentation: </strong>We present the case of an ICC patient with MSI-H who suffered rapid progression after a short-term remission with camrelizumab, a kind of PD-1 inhibitor, as second-line treatment. The patient's genomic and immune features were analyzed by next-generation sequencing and multiplex immunofluorescence staining to explore the possible mechanisms of the rapidly acquired resistance of ICIs in this MSI-H case.</p><p><strong>Conclusion: </strong>The genomic and immunohistochemical analysis showed that TGFBR2 mutation, loss of HLA B44 supertype, carrying B62 supertype, and increased PD-L1⁺ cells, macrophages, and Tregs in the tumor microenvironment might be related to the nonsustain benefit of ICIs in this MSI-H patient.</p>","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 3","pages":"281-288"},"PeriodicalIF":13.8,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a5/df/lic-2023-0012-0003-530273.PMC10427924.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10603731","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Drug-Off Criteria in Patients with Hepatocellular Carcinoma Who Achieved Clinical Complete Response after Combination Immunotherapy Combined with Locoregional Therapy.","authors":"Masatoshi Kudo","doi":"10.1159/000532023","DOIUrl":"10.1159/000532023","url":null,"abstract":"","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 4","pages":"289-296"},"PeriodicalIF":11.6,"publicationDate":"2023-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10601881/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71412862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Surgical Resection or Radiofrequency Ablation for Small Hepatocellular Carcinoma.","authors":"Qingbo Feng,&nbsp;Qiuping Ren,&nbsp;Jiaxin Li","doi":"10.1159/000527836","DOIUrl":"https://doi.org/10.1159/000527836","url":null,"abstract":"not required","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 2","pages":"178-179"},"PeriodicalIF":13.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a2/3a/lic-0012-0178.PMC10267511.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9654526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Atezolizumab and Bevacizumab Combination Therapy and Sequential Conversion Hepatectomy for Advanced Fibrolamellar Hepatocellular Carcinoma Presenting Pseudoprogression.","authors":"Ryota Matsuki,&nbsp;Naohiro Okano,&nbsp;Nobuhiro Hasui,&nbsp;Shohei Kawaguchi,&nbsp;Hirokazu Momose,&nbsp;Keiichiro Kitahama,&nbsp;Kiyotaka Nagahama,&nbsp;Masaharu Kogure,&nbsp;Yutaka Suzuki,&nbsp;Fumio Nagashima,&nbsp;Junji Shibahara,&nbsp;Hideaki Mori,&nbsp;Yoshihiro Sakamoto","doi":"10.1159/000527250","DOIUrl":"https://doi.org/10.1159/000527250","url":null,"abstract":"Fibrolamellar hepatocellular carcinoma (FLHCC) is a rare a rare subtype of hepatocellular carcinoma. The IMbrave150 trial demonstrated that atezolizumab and bevacizumab therapy (ABT) has better treatment outcomes than sorafenib for advanced HCC. However, since patients with known FLHCC were excluded from this trial, the effects of ABT on FLHCC remain unknown. We report the first case of ABT for advanced FLHCC followed by hepatectomy presenting pseudoprogression of lymph node (LN) metastases which was pathologically proven after surgery.\u0000The patient was a 30-year-old man with advanced FLHCC and multiple LN metastases behind the pancreatic head, and ABT was introduced. After four courses of treatment, CT indicated a minor decrease in the intratumor vascularity of the liver tumor. However, the size of metastatic LNs increased. Subsequently, the patient presented with bloody stool, and colonoscopy revealed immune-related colitis caused by atezolizumab. Therefore, the fifth course was canceled. A right hemihepatectomy following percutaneous transhepatic portal vein embolization (PTPE) was performed to increase the future liver remnant volume. After PTPE, dynamic CT revealed an objective response to ABT; SD in RECIST 1.1 (7% increase in the LN size and no change of liver tumor), and PR in modified RECIST (47% decrease in the intratumor vascularity of the liver tumor and LNs). Three weeks after PTPE, right hemihepatectomy plus nodal dissection was successfully performed. Pathological findings revealed that approximately 60%–70% of the liver tumor and 70%–80% of the metastatic LNs were necrotic, indicating a good response to ABT. The increasing size of metastatic LNs that occurred during the treatment course was deemed pseudoprogression. Pseudoprogression can be found in patients with solid malignancies treated with immune checkpoint inhibitors, however, rarely occurs in HCC. The first response to metastatic LNs was observed 20 weeks after ABT initiation combined with an increase in nodal volume and a decrease in vascularity. In the updated data of the IMbrave150 trial, 19% of the first responses occurred after week 24. Physicians should consider that ABT may also be effective in FLHCC and may cause pseudoprogression before determining a treatment strategy.","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 2","pages":"180-183"},"PeriodicalIF":13.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/48/7c/lic-0012-0180.PMC10267524.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9654525","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Imaging Diagnosis of Various Hepatocellular Carcinoma Subtypes and Its Hypervascular Mimics: Differential Diagnosis Based on Conventional Interpretation and Artificial Intelligence.","authors":"Yasunori Minami,&nbsp;Naoshi Nishida,&nbsp;Masatoshi Kudo","doi":"10.1159/000528538","DOIUrl":"https://doi.org/10.1159/000528538","url":null,"abstract":"<p><strong>Background: </strong>Hepatocellular carcinoma (HCC) is unique among malignancies, and its characteristics on contrast imaging modalities allow for a highly accurate diagnosis. The radiological differentiation of focal liver lesions is playing an increasingly important role, and the Liver Imaging Reporting and Data System adopts a combination of major features including arterial phase hyper-enhancement (APHE) and the washout pattern.</p><p><strong>Summary: </strong>Specific HCCs such as well or poorly differentiated type, subtypes including fibrolamellar or sarcomatoid and combined hepatocellular-cholangiocarcinoma do not often demonstrate APHE and washout appearance. Meanwhile, hypervascular liver metastases and hypervascular intrahepatic cholangiocarcinoma can demonstrate APHE and washout. There are still other hypervascular malignant liver tumors (i.e., angiosarcoma, epithelioid hemangioendothelioma) and hypervascular benign liver lesions (i.e., adenoma, focal nodular hyperplasia, angiomyolipoma, flash filling hemangioma, reactive lymphoid hyperplasia, inflammatory lesion, arterioportal shunt), which need to be distinguished from HCC. When a patient has chronic liver disease, differential diagnosis of hypervascular liver lesions can be even more complicated. Meanwhile, artificial intelligence (AI) in medicine has been widely explored, and recent advancement in the field of deep learning has provided promising performance for the analysis of medical images, especially radiological imaging data contain diagnostic, prognostic, and predictive information which AI can extract. The AI research studies have demonstrated high accuracy (over 90% accuracy) for classifying lesions with typical imaging features from some hepatic lesions. The AI system has a potential to be implemented in clinical routine as decision support tools. However, for the differential diagnosis of many types of hypervascular liver lesions, further large-scale clinical validation is still required.</p><p><strong>Key messages: </strong>Clinicians should be aware of the histopathological features, imaging characteristics, and differential diagnoses of hypervascular liver lesions to a precise diagnosis and more valuable treatment plan. We need to be familiar with such atypical cases to prevent a diagnostic delay, but AI-based tools also need to learn a large number of typical and atypical cases.</p>","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 2","pages":"103-115"},"PeriodicalIF":13.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4b/12/lic-0012-0103.PMC10267566.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9648639","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Phase 2 Study of the PD-1 Inhibitor Serplulimab plus the Bevacizumab Biosimilar HLX04 in Patients with Previously Treated Advanced Hepatocellular Carcinoma.","authors":"Zhenggang Ren,&nbsp;Guoliang Shao,&nbsp;Jie Shen,&nbsp;Li Zhang,&nbsp;Xu Zhu,&nbsp;Weijia Fang,&nbsp;Guoping Sun,&nbsp;Yuxian Bai,&nbsp;Jianbing Wu,&nbsp;Lianxin Liu,&nbsp;Yuan Yuan,&nbsp;Jingdong Zhang,&nbsp;Zhen Li,&nbsp;Ling Zhang,&nbsp;Tao Yin,&nbsp;Jincai Wu,&nbsp;Xiaoli Hou,&nbsp;Qingyu Wang,&nbsp;Jun Zhu,&nbsp;Jia Fan","doi":"10.1159/000526638","DOIUrl":"https://doi.org/10.1159/000526638","url":null,"abstract":"<p><strong>Introduction: </strong>Current treatments for patients with previously treated advanced hepatocellular carcinoma (HCC) provide modest survival benefits. We evaluated the safety and antitumor activity of serplulimab, an anti-PD-1 antibody, plus the bevacizumab biosimilar HLX04 in this patient population.</p><p><strong>Methods: </strong>In this open-label, multicenter, phase 2 study in China, patients with advanced HCC who failed prior systemic therapy received serplulimab 3 mg/kg plus HLX04 5 mg/kg (group A) or 10 mg/kg (group B) intravenously every 2 weeks. The primary endpoint was safety.</p><p><strong>Results: </strong>As of April 8, 2021, 20 and 21 patients were enrolled into groups A and B, and they had received a median of 7 and 11 treatment cycles, respectively. Grade ≥3 treatment-emergent adverse events were reported by 14 (70.0%) patients in group A and 12 (57.1%) in group B. Most immune-related adverse events were grade ≤3. The objective response rate was 30.0% (95% confidence interval [CI], 11.9-54.3) in group A and 14.3% (95% CI, 3.0-36.3) in group B. Median duration of response was not reached (95% CI, 3.3-not evaluable [NE]) in group A and was 9.0 months (95% CI, 7.9-NE) in group B. Median progression-free survival was 2.2 months (95% CI, 1.4-5.5) and 4.1 months (95% CI, 1.5-NE), and median overall survival was 11.6 months (95% CI, 6.4-NE) and 14.3 months (95% CI, 8.2-NE) in groups A and B, respectively.</p><p><strong>Conclusion: </strong>Serplulimab plus HLX04 showed a manageable safety profile and promising antitumor activity in patients with previously treated advanced HCC.</p>","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 2","pages":"116-128"},"PeriodicalIF":13.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4f/b3/lic-0012-0116.PMC10267516.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9648643","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Low Baseline CXCL9 Predicts Early Progressive Disease in Unresectable HCC with Atezolizumab Plus Bevacizumab Treatment.","authors":"Shunichi Hosoda,&nbsp;Goki Suda,&nbsp;Takuya Sho,&nbsp;Koji Ogawa,&nbsp;Megumi Kimura,&nbsp;Zijian Yang,&nbsp;Sonoe Yoshida,&nbsp;Akinori Kubo,&nbsp;Yoshimasa Tokuchi,&nbsp;Takashi Kitagataya,&nbsp;Osamu Maehara,&nbsp;Shunsuke Ohnishi,&nbsp;Akihisa Nakamura,&nbsp;Ren Yamada,&nbsp;Masatsugu Ohara,&nbsp;Naoki Kawagishi,&nbsp;Mitsuteru Natsuizaka,&nbsp;Masato Nakai,&nbsp;Kenichi Morikawa,&nbsp;Ken Furuya,&nbsp;Masaru Baba,&nbsp;Yoshiya Yamamoto,&nbsp;Kazuharu Suzuki,&nbsp;Takaaki Izumi,&nbsp;Takashi Meguro,&nbsp;Katsumi Terashita,&nbsp;Jun Ito,&nbsp;Takuto Miyagishima,&nbsp;Naoya Sakamoto","doi":"10.1159/000527759","DOIUrl":"https://doi.org/10.1159/000527759","url":null,"abstract":"<p><strong>Introduction: </strong>Atezolizumab plus bevacizumab treatment is highly effective in patients with unresectable hepatocellular carcinoma (HCC). However, progressive disease (PD) occurs in approximately 20% of HCC patients treated with atezolizumab plus bevacizumab, resulting in a poor prognosis. Thus, the prediction and early detection of HCC is crucial.</p><p><strong>Methods: </strong>Patients with unresectable HCC treated with atezolizumab plus bevacizumab and had baseline preserved serum (<i>n</i> = 68) were screened and classified according to their PD, 6 weeks after treatment initiation (early PD; <i>n</i> = 13). Of these, 4 patients each with and without early PD were selected for cytokine array and genetic analyses. The identified factors were validated in the validated cohort (<i>n</i> = 60) and evaluated in patients treated with lenvatinib.</p><p><strong>Results: </strong>No significant differences were observed in the genetic alterations in circulating tumor DNA. Cytokine array data revealed that baseline MIG (CXCL9), ENA-78, and RANTES differed substantially between patients with and without early PD. Subsequent analysis in the validation cohort revealed that baseline CXCL9 was significantly lower in patients with early PD than that in patients without early PD, and the best cut-off value of serum CXCL9 to predict early PD was 333 pg/mL (sensitivity: 0.600, specificity: 0.923, AUC = 0.75). In patients with lower serum CXCL9 (<333 pg/mL), 35.3% (12/34) experienced early PD with atezolizumab plus bevacizumab, while progression-free survival (PFS) was significantly shorter relative to that in patients without (median PFS, 126 days vs. 227 days; HR: 2.41, 95% CI: 1.22-4.80, <i>p</i> = 0.0084). While patients with objective response to lenvatinib had significantly lower CXCL9 levels compared with those of patients without.</p><p><strong>Conclusion: </strong>Baseline low serum CXCL9 (<333 pg/mL) levels may predict early PD in patients with unresectable HCC treated with atezolizumab plus bevacizumab.</p>","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 2","pages":"156-170"},"PeriodicalIF":13.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a3/a1/lic-0012-0156.PMC10267515.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9654524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Regression after Discontinuation of Regorafenib Administered for Sorafenib-Refractory Hepatocellular Carcinoma.","authors":"Heewon Bae,&nbsp;Je Ryung Gil,&nbsp;Moon Hyung Lee,&nbsp;Taekyu Lim","doi":"10.1159/000529139","DOIUrl":"https://doi.org/10.1159/000529139","url":null,"abstract":"A patient with hepatocellular carcinoma(HCC) staged Barcelona Clinic for Liver Cancer stage B started chemotherapy because of intrahepatic recurrence after hepatic segmentectomy. Regorafenib was started as a second-line treatment due to refractory to sorafenib treatment, but was discontinued after 26 months due to side effects. After two years of follow-up, the patients achieved unexpected long-term regression. We present the potential of regorafenib effect for anti-tumor immunity through possible pharmacological mechanisms.","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 2","pages":"184-187"},"PeriodicalIF":13.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ba/69/lic-0012-0184.PMC10267512.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9654528","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Regorafenib versus Cabozantinib as a Second-Line Treatment for Advanced Hepatocellular Carcinoma: An Anchored Matching-Adjusted Indirect Comparison of Efficacy and Safety.","authors":"Philippe Merle,&nbsp;Masatoshi Kudo,&nbsp;Stanimira Krotneva,&nbsp;Kirhan Ozgurdal,&nbsp;Yun Su,&nbsp;Irina Proskorovsky","doi":"10.1159/000527403","DOIUrl":"https://doi.org/10.1159/000527403","url":null,"abstract":"<p><strong>Introduction: </strong>The tyrosine kinase inhibitors regorafenib and cabozantinib remain the mainstay in second-line treatment of advanced hepatocellular carcinoma (HCC). There is currently no clear evidence of superiority in efficacy or safety to guide choice between the two treatments.</p><p><strong>Methods: </strong>We conducted an anchored matching-adjusted indirect comparison using individual patient data from the RESORCE trial of regorafenib and published aggregate data from the CELESTIAL trial of cabozantinib. Second-line HCC patients with prior sorafenib exposure of ≥3 months were included in the analyses. Hazard ratios (HRs) and restricted mean survival time (RMST) were estimated to quantify differences in overall survival (OS) and progression-free survival (PFS). Safety outcomes compared were rates of grade 3 or 4 adverse events (AEs), occurring in >10% of patients, and discontinuation or dose reduction due to treatment-related AEs.</p><p><strong>Results: </strong>After matching adjustment for differences in baseline patient characteristics, regorafenib showed a favorable OS (HR, 0.80; 95% CI: 0.54, 1.20) and ∼3-month-longer RMST over cabozantinib (RMST difference, 2.76 months; 95% CI: -1.03, 6.54), although not statistically significant. For PFS, there was no numerical difference in HR (HR, 1.00; 95% CI: 0.68, 1.49) and no clinically meaningful difference based on RMST analyses (RMST difference, -0.59 months; 95% CI: -1.83, 0.65). Regorafenib showed a significantly lower incidence of discontinuation (risk difference, -9.2%; 95% CI: -17.7%, -0.6%) and dose reductions (-15.2%; 95% CI: -29.0%, -1.5%) due to treatment-related AEs (any grade). Regorafenib was also associated with a lower incidence (not statistically significant) of grade 3 or 4 diarrhea (risk difference, -7.1%; 95% CI: -14.7%, 0.4%) and fatigue (-6.3%; 95% CI: -14.6%, 2.0%).</p><p><strong>Conclusion: </strong>This indirect treatment comparison suggests, relative to cabozantinib, that regorafenib could be associated with favorable OS (not statistically significant), lower rates of dose reductions and discontinuation due to treatment-related AEs, and lower rates of severe diarrhea and fatigue.</p>","PeriodicalId":18156,"journal":{"name":"Liver Cancer","volume":"12 2","pages":"145-155"},"PeriodicalIF":13.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/14/24/lic-0012-0145.PMC10267565.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9661031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}