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Novel biomarker-based model for the prediction of sorafenib response and overall survival in advanced hepatocellular carcinoma: a prospective cohort study

Title
Novel biomarker-based model for the prediction of sorafenib response and overall survival in advanced hepatocellular carcinoma: a prospective cohort study
Authors
Lee, Jeong-HoonCho, Young YounCho, Eun JuYu, Su JongKim, Yoon JunYoon, Jung-HwanKim, Hwi YoungLee, Dong Hyeon
Ewha Authors
김희영김휘영
SCOPUS Author ID
김휘영scopus
Issue Date
2018
Journal Title
BMC CANCER
ISSN
1471-2407JCR Link
Citation
vol. 18
Keywords
Hepatocellular carcinomaSorafenibResponseBiomarkerPrediction
Publisher
BIOMED CENTRAL LTD
Indexed
SCIE; SCOPUS WOS scopus
Abstract
Background: Prediction of the outcome of sorafenib therapy using biomarkers is an unmet clinical need in patients with advanced hepatocellular carcinoma (HCC). The aim was to develop and validate a biomarker-based model for predicting sorafenib response and overall survival (OS). Methods: This prospective cohort study included 124 consecutive HCC patients (44 with disease control, 80 with progression) with Child-Pugh class A liver function, who received sorafenib. Potential serum biomarkers (namely, hepatocyte growth factor [HGF], fibroblast growth factor [FGF], vascular endothelial growth factor receptor-1, CD117, and angiopoietin-2) were tested. After identifying independent predictors of tumor response, a risk scoring system for predicting OS was developed and 3-fold internal validation was conducted. Results: A risk scoring system was developed with six covariates: etiology, platelet count, Barcelona Clinic Liver Cancer stage, protein induced by vitamin K absence-II, HGF, and FGF. When patients were stratified into low-risk (score <= 5), intermediate-risk (score 6), and high-risk (score >= 7) groups, the model provided good discriminant functions on tumor response (concordance [c]-index, 0.884) and 12-month survival (area under the curve [AUC], 0.825). The median OS was 19.0, 11.2, and 6.1 months in the low-, intermediate-, and high-risk group, respectively (P < 0.001). In internal validation, the model maintained good discriminant functions on tumor response (c-index, 0.825) and 12-month survival (AUC, 0. 803), and good calibration functions (all P > 0.05 between expected and observed values). Conclusions: This new model including serum FGF and HGF showed good performance in predicting the response to sorafenib and survival in patients with advanced HCC.
DOI
10.1186/s12885-018-4211-2
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연구기관 > 의과학연구소 > Journal papers
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