, increasing the likelihood of potentially curative therapy and improved patient prognosis.
HCC accounts for the majority of primary liver cancers and mainly occurs in patients with cirrhosis or chronic hepatitis B virus infection. The prognosis of HCC is poor, largely owing to advanced disease stage at diagnosis.
The current guidelines recommend surveillance with twice-yearly liver ultrasound, with or without serum alpha-fetoprotein, for patients at risk for HCC, although the diagnostic performance is suboptimal.
The new “liquid biopsy” uses HCC-associated extracellular vesicles (EVs) to establish an HCC EV ECG score for distinguishing patients with early-stage HCC from at-risk controls with cirrhosis from a 400-mcL plasma sample.
“We’re the first team looking at extracellular vesicles as a detection biomarker for early-stage liver cancer, and our study showed it had outstanding performance,” study investigator Ju Dong Yang, MD, with Cedars-Sinai Medical Center, Los Angeles, said in a news release.
The study was published online in Hepatology.
In a phase 2 biomarker (case-control) study, the investigators tested their blood test in a training cohort of 106 individuals (45 patients with treatment-naive early-stage HCC and 61 with cirrhosis) and an independent validation cohort with 72 participants (35 patients with treatment-naive early-stage HCC and 37 with cirrhosis).
The HCC EV ECG score had “excellent accuracy” for discriminating between HCC and cirrhosis, with an area under the receiver operating characteristic curve (AUROC) of 0.95 and 0.93 in the training and validation cohorts, respectively, they report.
The diagnostic performance “remained excellent” among the subpopulations of HCC etiology and those with tumors within the Milan criteria.
Adding the serum alpha-fetoprotein level to the HCC EV ECG score did not improve its performance.
The researchers say that further validation of the blood test in a larger phase 2 study and a subsequent phase 3 study are needed to confirm its utility in clinical settings.
“We are planning on doing larger-scale studies to further validate this test and bring it into routine clinical practice here – and globally,” Dr. Yang said.
“In addition to its excellent performance, this marker has the advantages of being user friendly, cost efficient, and having a fast turnaround time – within 6 hours from sample collection to result,” Dan Theodorescu, MD, PhD, director of Cedars-Sinai Cancer, who is not an author on the study, said in the news release.
“Once this marker has been validated in subsequent studies, it can be easily adopted by existing PCR [polymerase chain reaction] facilities,” Dr. Theodorescu added.
The study was supported by an American College of Gastroenterology Junior Faculty Development Award, a Department of Defense Peer Reviewed Cancer Research Program Career Development Award, and the National Institutes of Health. Dr. Yang provides a consulting service for Exact Sciences, Gilead Sciences, and Eisai. Dr. Theodorescu reports no relevant financial relationships.
A version of this article first appeared on Medscape.com.