Backgrounds: There are limitations in using only radiological criteria to evaluate treatment outcomes in hepatocellular carcinoma (HCC). α-fetoprotein (AFP) is regarded as an indicator of tumour activity in HCC. Aims: We present a novel correlation between AFP response and survival outcome in patients treated with localized concurrent chemoradiotherapy (CCRT). Materials: From 2005 to 2008, 187 locally advanced HCC patients underwent localized CCRT (external beam radiotherapy at 45Gy over 5 weeks plus a concurrent hepatic arterial infusion of 5-fluorouracil during the first/fifth week), followed by repetitive hepatic arterial infusional chemotherapy (HAIC) with 5-fluorouracil and cisplatin. Among them, 149 with an elevated baseline AFP level (>20ng/ml) were finally studied. AFP response was defined as >50% decrease from baseline, 1 month after the completion of localized CCRT. Results: Patients' characteristics were as follows: median age (52 years); Child-Pugh class A/B (n=137/12 respectively); and portal vein thrombosis (n=118). AFP responders (101 patients) had better objective responses than AFP non-responders (48 patients) after CCRT (44.5 vs. 12.5%; P<0.001) and subsequent HAIC (51.5 vs. 16.7%; P<0.001). Both median progression-free survival (PFS, 8.1 vs. 3.9 months; P<0.001) and overall survival (OS, 13.3 vs. 5.9 months; P<0.001) were longer in AFP responders than AFP non-responders. In multivariate analysis, AFP response and objective response were independent factors affecting PFS and OS. Furthermore, AFP non-responders were more likely to have extrahepatic metastasis within 6 months of treatments initiation than AFP responders (59.5 vs. 25.9%; P<0.001). Conclusions: Early AFP response may be useful not only in predicting prognosis and treatment response but also in establishing optimized treatment plans for HCC.
All Science Journal Classification (ASJC) codes