Abstract
Management of advanced intrahepatic cholangiocarcinoma (iCCA) is challenging and overall survival is poor. Progress in the development of new therapeutic options for metastatic cholangiocarcinoma (CCA) has been slow; hence, to date, there are no approved second-line agents in this setting. Although the development of immune checkpoint inhibitors has significantly improved overall survival in a variety of malignancies, there has not been a clinically important impact in CCA. This report presents a 66-year-old patient with chemotherapy-refractory iCCA who experienced a prolonged response to immunotherapy. Tumor genome profiling revealed a high tumor mutation burden of 17 mutations per megabase in the absence of microsatellite instability. He was started on immunotherapy with nivolumab and has experienced an ongoing response for 16 months without clinical symptoms and only minimal radiologic disease.
MeSH terms
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Aged
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Antineoplastic Agents, Immunological / pharmacology
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Antineoplastic Agents, Immunological / therapeutic use*
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Antineoplastic Combined Chemotherapy Protocols / pharmacology
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Antineoplastic Combined Chemotherapy Protocols / therapeutic use
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Bile Duct Neoplasms / genetics
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Bile Duct Neoplasms / immunology
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Bile Duct Neoplasms / pathology
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Bile Duct Neoplasms / therapy*
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Bile Ducts, Intrahepatic / pathology
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Bile Ducts, Intrahepatic / surgery
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Capecitabine / pharmacology
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Capecitabine / therapeutic use
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Chemoradiotherapy, Adjuvant / methods
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Cholangiocarcinoma / genetics
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Cholangiocarcinoma / immunology
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Cholangiocarcinoma / secondary
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Cholangiocarcinoma / therapy*
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DNA Mutational Analysis
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Drug Resistance, Neoplasm / genetics
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Fluorouracil / pharmacology
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Fluorouracil / therapeutic use
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Humans
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Inguinal Canal / pathology
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Leucovorin / pharmacology
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Leucovorin / therapeutic use
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Liver / diagnostic imaging
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Liver / pathology
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Liver Neoplasms / secondary
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Liver Neoplasms / therapy*
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Lymphatic Metastasis / diagnostic imaging
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Lymphatic Metastasis / therapy*
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Male
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Microsatellite Instability
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Neoplasm, Residual
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Nivolumab / pharmacology
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Nivolumab / therapeutic use*
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Organoplatinum Compounds / pharmacology
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Organoplatinum Compounds / therapeutic use
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Positron Emission Tomography Computed Tomography
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Programmed Cell Death 1 Receptor / antagonists & inhibitors
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Treatment Outcome
Substances
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Antineoplastic Agents, Immunological
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Organoplatinum Compounds
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PDCD1 protein, human
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Programmed Cell Death 1 Receptor
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Nivolumab
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Capecitabine
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Leucovorin
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Fluorouracil