α‐fetoprotein response predicts survival benefits of thalidomide in advanced hepatocellular carcinoma

Abstract

Radiographic measurements do not always reflect the biological response of hepatocellular carcinoma to drug therapy. To evaluate the clinical implications of tumour marker (alpha-fetoprotein) response in advanced hepatocellular carcinoma patients with thalidomide treatment. Forty-two advanced hepatocellular carcinoma patients with baseline alpha-fetoprotein levels above 200 ng/mL and thalidomide therapy were included. Serum alpha-fetoprotein levels were measured every 4 weeks. alpha-fetoprotein response was defined as a 50% or greater reduction of alpha-fetoprotein levels for 4 or more weeks during treatment. Radiographic response was assessed by World Health Organization criteria; survivals were estimated by Kaplan-Meier method and prognostic factors were assessed by Cox's proportional hazard model. With intention-to-treat analysis, radiographic response and alpha-fetoprotein response were obtained in 7% (three of 42, 95% confidence interval: 0-15) and 24% (10 of 42, 95% CI: 10-38) of patients, respectively. All radiographic response was observed in alpha-fetoprotein responders. Multivariate analyses showed alpha-fetoprotein response was independent prognostic factor for both progression-free survival (relative risk = 0.394, 95% CI: 0.189-0.820, P = 0.013) and overall survival (relative risk = 0.241, 95% CI: 0.096-0.606, P =0.003), whereas radiographic response was not. alpha-fetoprotein response can more accurately reflect the biological response of advanced hepatocellular carcinoma to thalidomide therapy than radiographic response.