World’s First! Verified the Efficacy and Safety of Immune Checkpoint Inhibitor “Opdivo” in Advanced Hepatocellular Carcinoma Patients with Poor Liver Function -- Kindai University

The international collaborative research team centered around Prof. Masatoshi Kudo of Kindai University (Osaka Sayama City) Faculty of Medicine (Department of Gastroenterology and Hepatology) have verified for the first time in the world through a multi-national multi-institutional study that the efficacy and safety of “Nivolumab (Opdivo)”, immune checkpoint inhibitor, in advanced hepatocellular carcinoma (HCC) patients with mild to moderate impairment of liver function, was comparable to that of patients with good liver function. This research was published online in the “Journal of Hepatology”, an international medical journal publishing research and clinical studies in the field of hepatology, on Thursday, May 27, 2021 (9am, Japan time).

1. Key points

- Verified the efficacy of immune checkpoint inhibitor (Opdivo) in patients with advanced hepatocellular carcinoma (aHCC) and mild to moderate impairment of liver function (Child-Pugh B class).

- Kindai University School of Medicine led the international collaborative research for aHCC patients with liver dysfunction for which no treatment options were available before.

- Expected as a new therapeutic option for aHCC patients with poor liver functions.

2. Overview

For treating HCC, nine types of drugs are currently approved in the world (out of the nine, six types are approved in Japan): Five molecular targeted agents (lembatinib, sorafenib, cabozantinib, ramucirumab) and four immunotherapeutic agents (atezolizumab + bevacizumab, nivolumab, pembrolizumab, nivolumab + ipilimumab). However, these nine drugs have not been approved for use in aHCC patients with poor liver functions, and thus these drugs are available only for patients with good liver function (i.e. Child-Pugh A class). Systemic therapy for aHCC patients with liver dysfunction has been a major unmet need.

In this international collaborative research, in order to address this clinical problem, 49 aHCC patients with Child-Pugh B class were treated with immune checkpoint inhibitor “nivolumab (Opdivo)” and compared with 262 patients with good liver function (Child-Pugh A) to assess the efficacy and safety. As a result, it was verified for the first time in the world that the immunotherapy is effective and safe in Child-Pugh B aHCC patients, comparable to Child-Pugh A aHCC patients . In the future, nivolumab (Opdivo) is expected to be a novel treatment option for aHCC patients with poor liver function.

3. About publication

Research paper name: CheckMate 040 cohort 5: A phase I/II study of nivolumab in　patients with advanced hepatocellular carcinoma and Child-Pugh B cirrhosis

Journal name: Journal of Hepatology (Impact factor: 20.582＠2020) 

https://www.journal-of-hepatology.eu/article/S0168-8278(21)00313-5/fulltext