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Cholangiocarcinoma News

Important Advances in Cholangiocarcinoma Management

October 2020, Vol 1, No 2
Milind M. Javle, MD
Department of Gastrointestinal Medical Oncology
Division of Cancer Medicine
The University of Texas
M.D. Anderson Cancer Center
Houston, TX
NCI Task Force: Hepatobiliary Cancers

While the COVID-19 pandemic rages throughout the world, patients, providers, and healthcare advocates are learning to survive in an alternative (virtual) world that is likely to change the face of medicine forever. Cholangiocarcinoma (CCA) is a complex disease that requires multidisciplinary care. Virtual clinic visits and online tumor boards and symposia represent just a few of the countless innovations introduced over the past year.

In this issue of CCA News, we are highlighting topics presented at 2 important virtual conferences held this year—the Cholangiocarcinoma Foundation (CCF) conference held in July 2020, and the European Society for Medical Oncology (ESMO) conference held in September 2020. Below is a quick review of some of the topics discussed in this issue, with a focus on recent advances and ongoing challenges in CCA.

Timely diagnosis and treatment of CCA continue to present challenges. A survey of 700 patients diagnosed with CCA was discussed at the CCF conference, indicating that, on average, it took 22 months from the onset of CCA symptoms to the time the patient was diagnosed with and received treatment for CCA. Furthermore, on average, patients had to visit 2.5 oncologists before the diagnosis of CCA was established. These sobering statistics reflect the real-life scenario facing patients with this type of cancer.

This year, the CCF annual conference was chaired by Jesús M. Bañales, PhD, Group Leader, Liver Diseases Research Group, Biodonostia Institute, San Sebastián, Spain; Rachna T. Shroff, MD, MS, Chief, Section of GI Medical Oncology, University of Arizona Cancer Center, Tucson; and CCF CEO and Founder, Stacie C. Lindsey. Dr Bañales highlighted the importance of international collaboration in CCA, and Dr Shroff discussed the rapid changes in the evolution of therapies for CCA, with new targets such as HER2/neu and epigenetics.

The keynote address at the CCF conference was presented by William W. Li, MD, Chief Executive Officer, President, and Medical Director of the Angiogenesis Foundation, Cambridge, MA. In a particularly poignant introduction, Bruce Koppock, Vice Chair, CCF Board of Directors, introduced Dr Li to the attendees by highlighting how Dr Li had saved his life after his once-successful treatment for his CCA diagnosis was failing. Dr Li’s speech, titled “Dietary Modification of the Tumor Microenvironment: New Frontiers in Cancer Research and Therapy,” took a deep dive into how molecular nutrition and nutritional oncology will play critical roles in cancer medicine in the future.

Recently, ESMO released the first recommendations from a scientific society about the use of multigene next-generation sequencing (NGS). ESMO’s recommendations represent a key step in guiding clinicians toward a more conscious evidence-based use of multigene NGS in clinical practice. ESMO observed that although NGS has been transformative in cancer management, currently, testing is being ordered too late in the disease course. The availability of therapeutic options should guide the use of NGS, which remains expensive at this time.

The role of immunotherapy in CCA continues to be investigational. In an interesting late-breaking abstract presented at ESMO 2020, researchers discussed the results of a multicenter clinical trial of the combination of lenvatinib (Lenvima) and pembrolizumab (Keytruda) for the treatment of 187 patients with 6 different cancers, including CCA. The response rate was 10%, with a median duration of response of 5 months and a 68% disease control rate in a pretreated population of patients with biliary tract cancers. This study highlights the novel basket designs in clinical trials that may have a particular relevance to CCA.

The results of the FOENIX-CCA2 clinical trial of futibatinib used for the treatment of patients with CCA and FGFR2 fusions were presented at ESMO 2020. In this study, the overall response rate was 37.3%, with a median duration of response of 8.3 months. This study represents an important advance in the use of precision medicine in the treatment of patients with CCA.

Finally, in this issue we also feature the first “Guidelines for Management of Urgent Symptoms in Patients with Cholangiocarcinoma and Biliary Stents or Catheters Using the Modified RAND/UCLA Delphi Process,” which was initially published earlier in the year in Cancers and was developed by a team of international experts in association with the CCF.

If you have any questions, suggestions, or concerns, please address them to me at This email address is being protected from spambots. You need JavaScript enabled to view it..


Milind Javle, MD

Milind Javle, MD
Professor, Department of Gastrointestinal Medical Oncology
Division of Cancer Medicine
The University of Texas
MD Anderson Cancer Center
Houston, TX

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Incorporating FGFR Inhibitors into the Treatment Paradigm for Cholangiocarcinoma: Current Concepts and Future Directions
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On January 15, 2021, experts in the management of patients with cholangiocarcinoma (CCA) convened for a virtual accredited continuing education satellite symposium held during the 2021 annual meeting of the American Society of Clinical Oncology Gastrointestinal Cancers Symposium. The goal was to educate healthcare providers on various aspects of CCA, including epidemiology, current standards of care, unmet clinical needs, the safety and efficacy of fibroblast growth factor receptor (FGFR) inhibitors as second-line therapy, and practical approaches to incorporating FGFR inhibitors into the treatment paradigm for the disease.
Considering Toxicities Associated with Specific and Pan-FGFR Inhibitors
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Drs Javle, Báñales, and Hollebecque describe their thoughts about the use of pan-FGFR inhibitors compared with those that primarily target FGFR2 and the potential of treating CCA with inhibitors of FGFR1, 3, and 4. Moreover, they consider the most common adverse events associated with inhibitors of FGFR2, of which the most difficult to manage are hyperphosphatemia, nail toxicity, eye toxicity, and fatigue. The importance of educating oncologists on how to treat these toxicities is key to maintaining dose intensity of FGFR inhibitors.
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The Role of FGFR Inhibitors in Treating FGFR2 Fusion–Positive Cholangiocarcinoma
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Takeaways from the 2020 Cholangiocarcinoma Summit
By Milind M. Javle, MD
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In this last issue for the year, we are highlighting the proceedings of the Second Annual Cholangiocarcinoma (CCA) Summit, which was conducted on October 22-23, 2020. This summit attracted a wide, multidisciplinary audience and was very interactive, despite being a virtual event. An important focus for this meeting was the emergence of molecular targets in CCA and early successes seen with precision medicine in this disease. Many of the key presentations from the summit are included in the current issue and are highlighted below.

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