Therapeutic Effect of Regional Chemotherapy in Diffuse Metastatic Cholangiocarcinoma

SIMPLE SUMMARY: Cholangiocarcinoma are mostly diagnosed at a late stage and early recurrence is also very common with 5-year survival rates of <5% in unresectable, and 33% in resectable disease. Systemic therapy options are limited with unsatisfactory outcome. The aim of our study was to assess the efficacy of regional chemotherapy in diffuse metastatic cholangiocarcinoma. In 36 diffuse metastatic cholangiocarcinoma patients 189 cycles of regional chemotherapy using arterial infusion and perfusion techniques have been applied. Regional chemotherapy provided an excellent outcome with a median therapy specific survival of 12 months. Regional chemotherapy is effective and superior to current available and proposed therapy options in diffuse metastatic cholangiocarcinoma. ABSTRACT: Background: Current therapeutic options in diffuse metastatic cholangiocarcinoma (CCC) are limited with unsatisfactory results. We evaluated the efficacy of regional chemotherapy (RegCTx) using arterial infusion (AI), hypoxic stop-flow abdominal perfusion (HAP), upper abdominal perfusion (UAP) and isolated-thoracic perfusion (ITP) in 36 patients with metastatic perihilar and intrahepatic CCC. Methods: Ten patients had previously undergone a liver resection and in 14 patients the previous systemic chemotherapy (sCTx) approach had failed. A total of 189 RegCTx cycles (90 AI, 74 UAP, 13 HAP and 12 ITP) were applied using cisplatin alone or with Adriamycin and Mitomycin C. A minimum of three cycles were applied in 75% of the study population. The response was evaluated using RECIST criteria with MediasStat 28.5.14. Mortality, morbidity and survival analysis were performed using a prospective follow-up database and SPSS–28.0. Results: No procedure related mortality occurred. The overall morbidity was 56% and dominated by lymph fistulas at the inguinal access site. No grade III or IV haematological complication occurred. The overall response rate was 38% partial response, 41% stable and 21% progressive disease. Median overall survival was 23 months (95%CI 16.3–29.7). The RegCTx specific survival was 12 months (95%CI 6.5–17.5) in completely therapy naive patients but also in patients who had failed a sCTx attempt previously. Conclusion: RegCTx is feasible, safe and superior to the current proposed therapeutic options in metastatic CCC. The role of RegCTx should be determined in a larger cohort of diffuse metastatic CCC patients but also at early stages especially in initially not resectable but potentially resectable patients.