September/October 2021, Vol 2, No 3

Comprehensive molecular profiling has demonstrated a diverse landscape of oncogenic genomic alterations in cholangiocarcinoma (CCA), which are often the drivers of CCA. In a recent review article, Tanios S. Bekaii-Saab, MD, FACP, Vice Chair and Section Chief for Medical Oncology, Department of Internal Medicine, Mayo Clinic Cancer Center, Phoenix, AZ, and colleagues provided an overview of the molecular heterogeneity of CCA, discussing the role of molecular tests for the diagnosis of patients with intrahepatic CCA, and the implications of the genomic alterations in the treatment of patients with this aggressive disease.

On August 25, 2021, the FDA approved ivosidenib (Tibsovo; Servier Pharmaceuticals), an oral IDH1 inhibitor, for the treatment of adults with previously treated, locally advanced or metastatic cholangiocarcinoma (CCA) and an IDH1 mutation, as detected by an FDA-approved test. The FDA granted ivosidenib a priority review for this indication and an orphan drug designation.

This issue of CCA News features yet another exciting FDA approval for cholangiocarcinoma (CCA), ivosidenib, which was recently approved for CCA with IDH1 mutation. This is the third drug approved for CCA in the past 2 years, highlighting the critical role of molecular profiling in this cancer. Up to 20% of patients with intrahepatic CCA have IDH1 mutation, and the ClarIDHy phase 3 study demonstrated improvement in progression-free survival with ivosidenib versus placebo for chemorefractory disease. The FDA also approved the Oncomine Dx Target Test for IDH1 mutations.

I believe that the most powerful and positive force in medicine is that of a self-advocating patient. At the Cholangiocarcinoma Foundation, we strive to connect, educate, and empower patients with cholangiocarcinoma (CCA) to partner with their care team in navigating the course of their treatment. However, we understand that the treatment options are extremely limited for many patients with CCA.

Multimarker tumor panel testing is beneficial in helping to inform treatment choice in precision oncology. However, little is known about the clinic-based and organizational factors related to multimarker tumor panel testing, and why oncologists may not choose this testing.

Although cholangiocarcinoma (CCA) is a rare cancer that originates in the bile ducts, its incidence rate continues to rise in the United States, and many patients are diagnosed late, with unresectable tumor and poor prognosis. The majority of patients with extrahepatic CCA, including the perihilar subtype, require referral to a center with expertise in endoscopic retrograde cholangiopancreatography (ERCP) and interventional radiology, because of the complexities in obtaining a definitive diagnosis and durable biliary drainage.

Key topics related to intrahepatic cholangiocarcinoma (CCA) were presented at the 2021 Society of Interventional Oncology (SIO) and the Society of Interventional Radiology (SIR) meetings and were discussed at the CCA Summit. Bruno C. Odisio, MD, FSIR, Interventional Radiologist and Co-Director of Research, Interventional Radiology, M.D. Anderson Cancer Center, Houston, TX, reviewed the findings.

Interest among oncology specialists in cholangiocarcinoma (CCA) is on the rise, according to Flavio G. Rocha, MD, FACP, Director of Research, Digestive Disease Institute, and Associate Medical Director, Cancer Institute, University of Washington, Virginia Mason Medical Center, Seattle. At the CCA Summit, Dr Rocha reviewed hot topics in CCA that were presented at the 2021 Americas Hepato-Pancreato-Biliary Association (AHPBA) meeting.