Survival prediction for patients with non-resectable intrahepatic cholangiocarcinoma undergoing chemotherapy: a retrospective analysis comparing the tumor marker CA 19-9 with cross-sectional imaging

PURPOSE: Carbohydrate antigen (CA) 19-9 has been established as the main serum marker for patients with intrahepatic cholangiocarcinoma (ICC). The aim of this study was to compare the prognostic value of CA 19-9 changes versus response determined by imaging in patients with ICC undergoing chemotherapy. METHODS: Between 2003 and 2018, 151 patients with histopathologically confirmed ICC underwent chemotherapy at our tertiary care center for non-resectable or recurrent ICC, of whom 121 were included in this study. Serum CA 19-9 levels and imaging were retrospectively evaluated during chemotherapy. Log-rank testing and optimal stratification were used to classify patients into risk groups. RESULTS: Prior to chemotherapy, baseline serum CA 19-9 levels above the previously published cut-off of 37 U/ml were associated with poor survival (median OS 8.7 vs. 12.4 months, p = 0.003). After the beginning of chemotherapy, an increase in CA 19-9 of more than 40 U/ml resulted in impaired residual survival (median OS 5.0 vs. 12.1 months, p < 0.001). However, progressive disease at the first follow-up imaging proved the strongest predictor for poor outcome (median OS 4.6 vs. 15.5 months, p < 0.001). In contrast to prior studies, our data did not show statistically relevant differences in survival time with respect to absolute or relative decreases in serum CA 19-9 levels. CONCLUSION: In our study, the disease control rate—that is, the absence of progressive disease—was the strongest predictor of prolonged residual OS. To this end, both CA 19-9 changes and progressive disease on initial follow-up showed remarkable discriminatory power, with the latter slightly outperforming the former. Therefore, imaging should remain the mainstay of patient evaluation during follow-up. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s00432-020-03200-2) contains supplementary material, which is available to authorized users.