Safety and Efficacy of Low-Dose Regorafenib Combined with Nivolumab in Patients with Advanced HCC in Progression Beyond Two or More Lines of Tyrosine Kinase Inhibitors: Case Reportsand Short Review of the Literature

Authors

  • Olivier Rosmorduc Hepatology department, Centre hépato-biliaire, Paul Brousse Hospital, APHP, 94800 Villejuif, France and university Pierre and Marie Curie, Sorbonne University, Paris, France. Author
  • Pascal Hammel Department of Digestive and Medical Oncology, AP-HP, Paul Brousse Hospital, Villejuif, France.   Author
  • Audrey Coilly Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France. Author
  • Jean-Charles Duclos-Vallee Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France. Author
  • Emma Goldschmidt Department of Digestive and Medical Oncology, AP-HP, Paul Brousse Hospital, Villejuif, France. Author
  • Jamila Faivre Department of Medical and molecular Biology, Paul-Brousse University Hospital, AP-HP, Villejuif, France. Author
  • Alina Pascale Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France. Author
  • Antoinette Lemoine-Corbel Department of Medical and molecular Biology, Paul-Brousse University Hospital, AP-HP, Villejuif, France. Author
  • Catherine Guettier Department of Pathology, AP-HP Bicêtre Hospital, France, University Paris Sud, University Paris Saclay, France. Author
  • Maite Lewin Department of Radiology, AP-HP, Paul Brousse Hospital, Villejuif, France. Author
  • Astrid Laurent Bellue Department of Pathology, AP-HP Bicêtre Hospital, France, University Paris Sud, University Paris Saclay, France. Author
  • Yasmina Ben Merabet Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France. Author
  • Didier Samuel University Paris-Sud, UMR-S 1193, University Paris-Saclay, Inserm-Paris Sud research Unit 1193, University Paris-Saclay; Hepatinov, Villejuif, France. Author

DOI:

https://doi.org/10.47363/JDCRS/2024(5)146

Keywords:

Hepatocellular Carcinoma, Checkpoint Inhibitor, Tyrosine Kinase Inhibitor, Nivolumab, Regorafenib

Abstract

Background and Aims: A few clinical studies have suggested that low doses of anti-angiogenic molecules might enhance the therapeutic effects of anti-PD1 in HCC and may improve their tolerance. Here, we present case reports of patients treated with low-dose regorafenib combined with nivolumab after the failure of at least two lines of oral chemotherapy.

Design: We report 12 observations of patients with advanced HCC in progression after at least two lines of tyrosine kinase inhibitor (TKI) therapy who received regorafenib (starting with 80 mg/day) combined with nivolumab (3 mg/kg/14 days/IV) until progression or unacceptable toxicity.The safety based on NCI-CTCAE 4 and the clinical efficacy (progression free survival, overall response rate, disease control rate, time to treatment response and median duration of response) were retropectively assessed.

Results: Median age was 68 years (IQR [64; 71.5]), and 75% had BCLC-C. The median follow-up was 18.8 months (IQR [11.5; 23.4]) and the median duration of treatment was 10 months (IQR [3.8 ;17]). Because of the frequent early adverse events associated with regorafenib, this treatment remained at an initial dose of 80 mg/day in all patients. Of note, most grade III adverse events (AEs) occurred later after a median duration of combined treatment of 10.5 months (IQR ), were also mainly due to regorafenib, but allowed the pursuit of nivolumab alone [5,11,5]. The therapeutic combination led to a partial response in three patients (30 %) and a stable disease in two patients (20 %), corresponding to a disease control rate of 50% at 12 months in the 10 patients who were trated more than 2 months. The median TTR was 2 months (IQR) [1-9]. The median duration of disease control and response was 22 (IQR [21;
24] ) and 24 months (IQR), respectively [23-25].

Conclusion: Combination of low-dose regorafenib and nivolumab might be associated with an interesting disease control rate and manageable safety profile in patients with progressive advanced HCC beyond the second-line treatment with tyrosine kinase inhibitors.

Author Biographies

  • Olivier Rosmorduc, Hepatology department, Centre hépato-biliaire, Paul Brousse Hospital, APHP, 94800 Villejuif, France and university Pierre and Marie Curie, Sorbonne University, Paris, France.

    Olivier Rosmorduc,Hepatology department, Centre hépato-biliaire, Paul Brousse Hospital, APHP, 94800 Villejuif, France and university Pierre and Marie Curie, Sorbonne University, Paris, France.

  • Pascal Hammel, Department of Digestive and Medical Oncology, AP-HP, Paul Brousse Hospital, Villejuif, France.  

    Pascal Hammel,Department of Digestive and Medical Oncology, AP-HP, Paul Brousse Hospital, Villejuif, France.  

  • Audrey Coilly, Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France.

    Audrey Coilly,Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France.

  • Jean-Charles Duclos-Vallee, Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France.

    Jean-Charles Duclos-Vallee,Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France.

  • Emma Goldschmidt, Department of Digestive and Medical Oncology, AP-HP, Paul Brousse Hospital, Villejuif, France.

    Emma Goldschmidt,Department of Digestive and Medical Oncology, AP-HP, Paul Brousse Hospital, Villejuif, France.

  • Jamila Faivre, Department of Medical and molecular Biology, Paul-Brousse University Hospital, AP-HP, Villejuif, France.

    Jamila Faivre,Department of Medical and molecular Biology, Paul-Brousse University Hospital, AP-HP, Villejuif, France.

  • Alina Pascale, Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France.

    Alina Pascale,Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France.

  • Antoinette Lemoine-Corbel, Department of Medical and molecular Biology, Paul-Brousse University Hospital, AP-HP, Villejuif, France.

    Antoinette Lemoine-Corbel,Department of Medical and molecular Biology, Paul-Brousse University Hospital, AP-HP, Villejuif, France.

  • Catherine Guettier, Department of Pathology, AP-HP Bicêtre Hospital, France, University Paris Sud, University Paris Saclay, France.

    Catherine Guettier,Department of Pathology, AP-HP Bicêtre Hospital, France, University Paris Sud, University Paris Saclay, France.

  • Maite Lewin, Department of Radiology, AP-HP, Paul Brousse Hospital, Villejuif, France.

    Maite Lewin,Department of Radiology, AP-HP, Paul Brousse Hospital, Villejuif, France.

  • Astrid Laurent Bellue, Department of Pathology, AP-HP Bicêtre Hospital, France, University Paris Sud, University Paris Saclay, France.

    Astrid Laurent Bellue,Department of Pathology, AP-HP Bicêtre Hospital, France, University Paris Sud, University Paris Saclay, France.

  • Yasmina Ben Merabet, Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France.

    Yasmina Ben Merabet,Department of Hepatology, Hépato-Biliary center, AP-HP Paul Brousse Hospital, Villejuif, France.

  • Didier Samuel, University Paris-Sud, UMR-S 1193, University Paris-Saclay, Inserm-Paris Sud research Unit 1193, University Paris-Saclay; Hepatinov, Villejuif, France.

    Didier Samuel,University Paris-Sud, UMR-S 1193, University Paris-Saclay, Inserm-Paris Sud research Unit 1193, University Paris-Saclay; Hepatinov, Villejuif, France.

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Published

2024-07-15