Cholangiocarcinoma

Of the approximately 18,000 individuals who are diagnosed annually in the United States with biliary cancer, an estimated 8000 are diagnosed with cholangiocarcinoma (CCA), and the 5-year survival rate is less than 20%. CCA is often diagnosed at advanced stages when treatment is only minimally effective.
Barcelona, Spain—Alterations in the fibroblast growth factor receptor (FGFR2)2 gene have been identified as driver mutations in cholangiocarcinoma (CCA). Durable objective responses were observed in >33% of patients with locally advanced or metastatic CCA and FGFR2 rearrangements or fusions who received treatment with pemigatinib, a selective oral inhibitor of FGFR1, FGFR2, and FGFR3. Data from the single-arm, open-label phase 2 clinical trial FIGHT-202, which was presented at the ESMO Congress 2019, revealed that investigational pemigatinib induced a response in 35.5% of the 107 patients with FGFR2 fusions or rearrangements (cohort A), with a median duration of response of 7.5 months.
Barcelona, Spain—Ivosidenib (Tibsovo), an oral therapy that targets isocitrate dehydrogenase-1 (IDH1) mutation, significantly improved pro­gression-free survival (PFS) in patients with advanced cholangiocarcinoma (CCA) and an IDH1 mutation, in a phase 3 clinical trial reported lead investigator Ghassan K. Abou-­Alfa, MD, Medical Oncologist, Gastrointestinal Oncology Service, Memorial Sloan Kettering Cancer Center, New York City, at the ESMO Congress 2019.
Diagnosis of cholangiocarcinoma (CCA) is often identified at a late stage. Analyzing the tumor­specific mutation profile of a patient with CCA can improve the diagnosis and treatment for the individual patient.

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