New data from two clinical trials that were presented at the International Liver Congress 2013 in Amsterdam, the Netherlands, demonstrated substantial improvements in the detection of both hepatocellular carcinoma (HCC) and cholangiocarcinoma using diagnostic urine tests.

HCC is common throughout the world and most often develops as a late complication of chronic viral hepatitis or cirrhosis of any cause. Because the overall survival rate of HCC is poor, screening for HCC offers the best hope for early detection, eligibility for treatment, and improved survival. While effective therapies exist, the available screening tests to detect HCC, which are alpha-fetoprotein (AFP) and ultrasound, are reported to have low sensitivity and specificity (50%–85% and 70%–90%, respectively).

Preliminary data demonstrate the performance of urinary metabolites in helping to diagnose HCC. Urine samples were collected from four subject groups in West Africa on the case-control platform of PROLIFICA ‘Prevention of Liver Fibrosis and Carcinoma in Africa’ as follows: patients with HCC (n=65), cirrhosis (n=36), noncirrhotic liver disease (n=110), and healthy controls (n=91). HCC patients were diagnosed using guidelines from the European Association for the Study of the Liver (EASL).

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Multivariate analyses of urinary nuclear magnetic resonance (NMR) spectra showed a distinct profile for urine of patients with HCC compared to the other conditions, and the urinary metabolite profile outperformed serum AFP.

“These findings will be welcomed by physicians as they validate urinary metabolic profiling as a potential screening tool for HCC, with superior diagnostic accuracy to serum AFP and, if investigated further and put into practice, this noninvasive technique could simplify and improve clinical diagnosis and outcomes for patients,” said Mark Thursz, MD, Secretary General of EASL.

Similarly, detection of cholangiocarcinoma remains a diagnostic challenge and physicians will be encouraged by results from a Phase II study showing that a combined bile and urine proteomic test increased diagnostic accuracy of cholangiocarcinoma in patients with biliary strictures (an abnormal narrowing of the common bile duct) of unknown origin.

Researchers recently established diagnostic peptide marker models in bile and urine to detect both local and systemic changes during cholangiocarcinoma progression, and so they combined both models with the aim of reaching a higher diagnostic accuracy.

The data demonstrated this model enables impressive cholangiocarcinoma diagnosis with an accuracy of more than 90% that is most applicable for patients with biliary strictures of unknown origin referred to endoscopy.

Thursz added, “These important findings substantially improve the diagnosis of [cholangiocarcinoma] and may lead to early therapy and improved prognosis. Overall both data sets demonstrate the increasing value of proteomic and metabonomic techniques and if confirmed by further investigation, clinicians may soon be using simple urine dip-stick tests to diagnose HCC and [cholangiocarcinoma].”

Cholangiocarcinoma or bile duct cancer is rare and almost always adenocarcinoma which starts in the lining of the bile duct. The cause of most cholangiocarcinomas is unknown but people with chronic inflammatory bowel conditions or congenital abnormalities of the bile duct have a higher risk of developing the cancer.