A monocentric real-word experience of CT-IO combination in advanced BTC.

Abstract

e16247 Background: Biliary tract neoplasms are very aggressive, but new frontiers of treatment are in course of developing for advanced disease. One of the most important clinical trials in this setting was the TOPAZ-1 trial, which led to the approval of the new standard of first-line therapy with the combination of cisplatin, gemcitabine and durvalumab. Methods: We conducted the analysis of real-life data relating to the association CT-IO in advanced biliary tract cancers of 45 patients who benefited from this innovative combination therapy at the Medical Oncology Unit of the University of Naples "Federico II", between December 2021 and August 2024, and whose characteristics are summered into Figure 1. Results: The ORR was 35%, while the DCR was 57%. The median follow-up of the present analysis was about 16 months (479 days) and during this time frame the 38% of patients died, 2 because of complications unrelated to CT-IO received and 15 for tumour progression.The mOS was 14.9 months and the mPFS was 8 months. The primary tumour site was found to be located at the level of the intrahepatic biliary tract in 60% of cases, while extrahepatic cholangiocarcinoma corresponded to 31% and cholecystic neoplasms to 9% of the total. The most important side effects experienced by the patients examined were as follows: nausea G1 (48.89%) and vomiting G1 (33.33%), diarrhoea G1 (35.56%) and G2 (4.44%), hypertransaminasemia G1 (42.22%), hyperbilirubinemia G1 (44.44%) and G2 (8.89%), anaemia G1 (48.89%), G2 (6.67%) and G3 (2.22%), neutropenia G1 (15.56%), G2 (20%) and G3 (4.44%), thrombocytopenia G1 (24.44%) and G2 (11.11%) and asthenia (51.11%) and G2 (28.89%). Regarding the immune-related side effects, hypothyroidism, both grade 1 and grade 2, occurred in 2.22% of cases. Conclusions: Our single-centre Italian experience reflected the results of the TOPAZ-1 study and the most recent analysis of clinical practice data from Europe, USA and Asia published in September 2024. The set of data obtained confirmed the robustness of the new standard of therapy, thanks to its efficacy and tolerability demonstrated. Table of patient characteristics, N (%) = 45. Label N (%) N= 45 Sex  M 28 (62%)  F 17 (38%) Age at diagnosis 65 (range 31-82) Tobacco  yes 11 (25%)  not 11 (25%)  ex- 11 (25%) BMI  underweight 4 (8,89%)  normal 14 (31,11%)  overweight 19 (42,22%)  obesity grade 1 7 (15,76%)  obesity grade 2 1 (2,22%) Site of primitif tumour  intrahepatic 26 (60%)  extrahepatic 15 (31%)  gallbladder 4 (9%)  Hepatitis 12 (25%)  HBV+ 6 (50%)  HCV + 6 (50%) Stage of disease  socally advanced 24 (53,3%)  methastatic 21 (46,7%) ECOG PS  0 39 (86,7%)  1 6 (13,3%) Median CA 19-9 (UI/mL)  in normal range 23 (51,11%)  > of normal range 22 (48,89%)