Conversion therapy in advanced perihilar cholangiocarcinoma based on patient-derived organoids: A case report

Patient-derived organoids (PDOs) have been demonstrated to predict the response to drugs in multiple cancer types. However, it remains unclear about its application in cholangiocarcinoma.

A 59-year-old woman was admitted to the hospital due to upper abdominal pain for over 8 months. According to relevant examinations, she was diagnosed as perihilar cholangiocarcinoma (pCCA) with intrahepatic metastasis and perihilar lymphatic metastasis. After multidisciplinary team discussion, percutaneous transhepatic cholangiodrainage was performed to relieve biliary obstruction, and puncture biopsy was conducted to confirm the pathological diagnosis. Transarterial chemoembolization with nab-paclitaxel was used in combination with toripalimab and lenvatinib, but the levels of tumor markers including alpha fetal protein, carcinoembryonic antigen, carbohydrate antigen 15-3 and cancer antigen 125 were still raised. The PDO for drug screening showed sensitive to gemcitabine and cisplatin. Accordingly, the chemotherapy regimen was adjusted to gemcitabine and cisplatin in combination with toripalimab and lenvatinib. After 4 cycles of treatment, the tumor was assessed resectable, and radical surgical resection was performed successfully. One year after surgery, the patient was still alive, and no recurrence or occurred.

PDOs for drug sensitivity contribute to screening effective chemotherapy drugs for advanced pCCA, promoting conversion therapy and improving the prognosis.