Abstract
The need to estimate prognosis of advanced BTC (aBTC) patients treated with first-line chemotherapy is compelling. The aim of the study is to evaluate the ECSIPOT (psECogSIiPnigOT) index, influenced by PECS (PsECogSii) index, prognostic nutritional index (PNI), and GOT. This international study was conducted on a training cohort of 126 patients and in three validation cohorts, both European and Korean. ECSIPOT index formula: (PECS:0 = 1 point; PECS:1 = 1.4 points; PECS:2 = 3.2 points) + (PNI > 36.7 = 1 point; PNI < 36.7 = 2 points) + (GOT < 100 = 1 point; GOT > 100 = 2 points). Event-time distributions were estimated using the Kaplan-Meier method, and survival curves were compared using the log-rank test. In the training cohort, mOS was 12.9, 6.3, and 2.8months for patients with ECSIPOT-0, ECSIPOT-1, and ECSIPOT-2, respectively (ECSIPOT-0: HR 1; ECSIPOT-1: HR 2.11; ECSIPOT-2: HR 4.93; p < 0.0001). In the first validation cohort, mOS was 11.5, 7.3, and 3.3months for ECSIPOT-0, ECSIPOT-1, and ECSIPOT-2, respectively (ECSIPOT-0: HR 1; ECSIPOT-1: HR 1.74; ECSIPOT-2: HR 3.41; p < 0.0001). In the second validation cohort, mOS was 25.2, 12.5, and 3.0months for ECSIPOT-0, ECSIPOT-1, and ECSIPOT-2, respectively (ECSIPOT-0: HR = 1; ECSIPOT-1: HR 2.33; ECSIPOT-2: HR 8.46; p < 0.0001). In the third validation cohort, mOS was 11.8, 8.1, and 4.6months for ECSIPOT-0, ECSIPOT-1, and ECSIPOT-2, respectively (ECSIPOT-0: HR = 1; ECSIPOT-1: HR 1.47; ECSIPOT-2: HR 3.17; p < 0.0001). Multivariate analysis in all cohorts confirmed the ECSIPOT index as an independent prognostic factor for OS. The easy assessment and good risk-stratification performance make the ECSIPOT index a promising tool to comprehensively estimate the prognosis of aBTC patients.
Highlights
Intrahepatic cholangiocarcinoma is a rare and aggressive liver neoplasm originating from the cholangiocytes of the intrahepatic biliary duct branches tract [1].Its incidence accounts for 10–20% of all biliary tract carcinoma (BTC), and its prognosis is dismal; surgery is potentially curative only at the early stage, while survival for the advanced intrahepatic cholangiocarcinoma (iCCA) patients reaches 5 years in less than 10% [2]
Cells were isolated after the dissociation of the tumor mass derived from a 4th generation patient-derived xenograft (PDX)
Preclinical models are limited, and the in vitro models are mainly represented by cell lines derived from Eastern patients
Summary
Intrahepatic cholangiocarcinoma (iCCA) is a rare and aggressive liver neoplasm originating from the cholangiocytes of the intrahepatic biliary duct branches tract [1].Its incidence accounts for 10–20% of all biliary tract carcinoma (BTC), and its prognosis is dismal; surgery is potentially curative only at the early stage, while survival for the advanced iCCA patients reaches 5 years in less than 10% [2]. Clinical data indicated that patients treated with gemcitabine-cisplatin regimen as first-line, followed by fluoropyrimidine-based chemotherapy as second-line showed an overall response rate of 3% and a median progression-free survival of 1.9 months (95% confidence interval (CI), 1.6–2.2) [8]. These data may suggest a cross-resistance to both drugs, as already demonstrated in preclinical models of pancreatic cancer and BTC [9,10]. It is mandatory to find alternative therapeutic options for these multidrug-resistant patients
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