Abstract
Intrahepatic cholangiocarcinoma (ICC) is an aggressive biliary tract cancer (BTC) with distinct anatomic, molecular, and clinical characteristics. Over the last 10-20 years, ICC has become the focus of increasing concern largely due to its rising incidence and high mortality rates in various parts of the world, including the United States.Surgery is the only potentially curative treatment option for ICC; however, recurrence rate is high, and prognosis is poor in patients with recurrent disease. The chemotherapy regimen of gemcitabine-cisplatin (GemCis) is still the standard of care for patients with unresectable metastatic ICC. There is limited data regarding pathologic ICC response to palliatively intentioned systemic treatment.Here, we report a case of a 47-year-old Caucasian male with metastatic ICC microsatellite stable (MSS) and TMB 49 mutation per megabase who achieved complete pathological response with sequential GemCis/nab-paclitaxel and pembrolizumab. This case highlights the effect of sequential neoadjuvant chemoimmunotherapy in a patient with high tumor mutational burden (TMB-H) ICC, emphasizing the importance of molecular testing, which provides valuable information that can be used in clinical practice to better select targeted chemotherapy regimens.
Highlights
Complete surgical resection remains a potentially curative option for intrahepatic cholangiocarcinoma (ICC); only about a third of patients diagnosed with ICC qualify for surgery [1], despite having a poor prognosis with a high rate of recurrence that requires a more aggressive therapeutic intervention [2,3]
Approved treatments for patients with unresectable ICC are limited to chemotherapy regimens tested in heterogeneous study populations of patients with biliary tract cancer (BTC)
We described a unique patient with metastatic TMB-H ICC who achieved a complete pathological response to sequential chemo- and immunotherapy
Summary
Complete surgical resection remains a potentially curative option for intrahepatic cholangiocarcinoma (ICC); only about a third of patients diagnosed with ICC qualify for surgery [1], despite having a poor prognosis with a high rate of recurrence that requires a more aggressive therapeutic intervention [2,3]. 12 patients were able to convert to resectable disease and undergo surgery, two of whom achieved a pathological complete response (pCR) These findings, while promising, still need to be corroborated in the ongoing phase III trial led by the same group (NCT03768414) [5]. Pembrolizumab is an anti-PD-1 antibody approved by the US Food and Drug Administration for the treatment of many cancer types as well as agnostic tumors for patients with microsatellite instability, mismatch repair deficiency, and for which an effective response was recently observed [8,9]. Follow-up CT scans showed a radiologic response as the tumor size decreased from 10x8.3 cm to 6.2x4.3 cm within three months of starting immunotherapy (Figure 1B). Pathologic analysis confirmed that the patient underwent a complete response to treatment, showing no viable tumor in the specimen (Figure 7). IVC: inferior vena cava; RPV: right portal vein; HA: hepatic artery; RHV: right hepatic vein
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