Study May Have Good News for Patients With Early-stage Liver Cancer 

Immunotherapy may make surgery an option for patients who would be considered ineligible under traditional rules. 

Surgery is usually the best treatment for early-stage hepatocellular carcinoma, the most common type of liver cancer, but only a minority of patients qualify for an operation. New research, however, indicates that a course of immunotherapy may make surgery available to more patients with high-risk cancers, with outcomes similar to those with less complex cases. The study was published in the American Association for Cancer Research (AACR) journal Cancer Research Communications.

“There is a group of patients with high-risk liver cancer who, in a contemporary era, may have long-term survival through aggressive treatment with systemic therapy followed by surgery,” said Mark Yarchoan, MD, senior author of the study and an associate professor of oncology at the Johns Hopkins Kimmel Cancer Center in Baltimore.

Treatment of early-stage hepatocellular carcinoma is often complicated by the presence of more than one tumor, proximity to major blood vessels, or invasion of blood vessels by the cancer. Because of these and other factors, only about 30% of hepatocellular carcinoma patients are eligible for surgery to remove their tumors, according to Mari Nakazawa, MD, a clinical research fellow at the Johns Hopkins Kimmel Cancer Center and first author of the study. 

“There’s a strong unmet need to expand the number of patients who may be eligible for surgery and, further, to transform more patients with early-stage liver cancer into long-term survivors of this disease,” Dr. Nakazawa said. 

In the study, researchers looked at 92 cases in which patients had surgery for hepatocellular carcinoma at Johns Hopkins Hospital from 2017 to 2023. Of the 92 patients, 36 received a type of immunotherapy called immune checkpoint inhibitors (ICI) before the surgery. Many of these patients were part of clinical trials evaluating ICI therapy before surgery, and most would not have been eligible for surgery if they had not been in the clinical trials.  

The study found that patients who received checkpoint inhibitors before surgery had outcomes that compared well to the patients who went straight to surgery. This was despite the fact that 61.1% of the patients receiving presurgery ICIs also had high-risk features that would usually lead to worse outcomes.  

Nevertheless, 94.4% of patients who received ICI therapy had successful, margin-negative surgery.  

“This study shows that the criteria by which we classify patients as being candidates for curative therapy is probably too narrow for this disease,” Dr. Yarchoan said. 

The authors noted that the study was retrospective in that it relied on data from previously conducted studies. They said the study could provide hypotheses that could be tested in forward-looking studies.  

“Prospective trials that are thoughtfully designed in the right populations can help us understand which patients can benefit most from this approach,” Dr. Nakazawa said. 

“Our findings demonstrate that systemic therapy may not only be useful for patients with advanced disease but can potentially be paradigm changing in patients with early-stage disease,” Dr. Yarchoan added.