Bernard Paule¹, Paola Andreani¹, Marie-Pierre Bralet², Catherine Guettier^3-5, René Adam^1,6,7, Denis Castaing^1,6,7 and Daniel Azoulay^1,6,7

¹AP-HP Hôpital Paul Brousse, Centre Hépato-Biliaire, Villejuif F-94800, France. ²Inserm UMR-S775, Université  Paris-Descartes. ³AP-HP Hôpital Paul Brousse, Anatomie Pathologique, Villejuif F-94800, France. ⁴Université Paris-Sud, UMR-S 785, Villejuif F-94800, France. ⁵Inserm, Unité 785, Villejuif F-94800, France. ⁶Université Paris-Sud, UMR-S 602, Villejuif F-94800, France. ⁷Inserm, Unité 602, Villejuif F-94800, France.

Abstract

Background: There is no standard adjuvant chemotherapy to prevent recurrent cholangiocarcinoma (CCA), a rare cancer with poor prognosis. We assessed the efficacy and safety of GEMOX on intrahepatic and hilar CCA with high-risk factors after curative surgery.

Patients and Methods: Twenty two patients (mean age: 57 years old) with CCA received 6 cycles of GEMOX: gemcitabine 1,000 mg/m² on day 1 and oxaliplatin 85 mg/m² on day 2, q3w after a curative surgery.

Results: All patients completed 6 cycles of GEMOX. EGFR membranous expression was present in 20 CCA. The 5-year survival rate was 56% (CI 95%: 25.7–85.4); 2-year disease free survival rate was 28% (CI 95%: 3.4–52.6). Median time to progression was 15 months. The rate of recurrence after surgery and chemotherapy was 63% (14/22). Two patients died of disease progression. Twelve patients received cetuximab/GEMOX at the time of relapse. Six died after 12 months (9–48 months), three are still alive suggesting a clinical applicability of EGFR inhibitors in CCA.

Conclusion: Adjuvant chemotherapy with GEMOX alone seems ineffective in intrahepatic and hilar CCA with a high risk of relapse. Additional studies including targeted therapies to circumvent such poor chemosensitivity are needed.