1*Department of Internal Medicine, UT Southwestern Medical Center and Parkland Health Hospital System †Department of Surgery ‡Harold C. Simmons Cancer Center, UT Southwestern Medical Center, Dallas, TX §VA Center for Clinical Management Research ∥Department of Internal Medicine, University of Michigan Health System, Ann Arbor, MI.
Surveillance for hepatocellular carcinoma (HCC) is recommended in patients with cirrhosis; however, early detection efforts are limited by suboptimal effectiveness.
To derive and validate a model to accurately distinguish cirrhotic patients with and without HCC and compare the accuracy of the model to that of α-fetoprotein (AFP) alone.
We conducted a case-control study of cirrhotic patients with and without HCC seen at a large urban hospital system between January 2005 and June 2012. We derived multivariate logistic regression models for the presence of HCC and early-stage HCC. Discriminatory power was evaluated using receiver operating characteristic curve analysis in derivation and validation cohorts using a 10-fold cross-validation approach.
We identified 1356 patients with cirrhosis, with (n=455, 147 early stage) and without (n=901) HCC. We found that AFP>20 ng/mL and FIB-4, a noninvasive marker of fibrosis, were significantly associated with the presence of HCC (OR=10.5; 95% CI, 7.9-13.9 and OR=1.05; 95% CI, 1.03-1.07, respectively) and early-stage HCC (OR=4.4; 95% CI, 2.9-6.5 and OR=1.06; 95% CI, 1.03-1.09, respectively). Models incorporating AFP and FIB-4 had good discriminatory power, with c-statistics of approximately 0.80, in both derivation and validation cohorts. The model for early-stage HCC had higher discriminatory power than AFP alone (c-statistic 0.73; 95% CI, 0.69-0.78) in derivation and validation cohorts (P=0.02 and 0.15, respectively).
Models including AFP and FIB-4 can accurately discriminate cirrhotic patients with early-stage HCC from those without HCC.