Abstract
It is not clear whether survival times of patients with hepatocellular carcinoma (HCC) are associated with their response to therapy. We analyzed the association between tumor response and survival times of patients with HCC who were treated with locoregional therapies (LRTs) (chemoembolization and radioembolization). Patients received LRTs over a 9-year period (n = 463). Patients with metastases, portal venous thrombosis, or who had received transplants were excluded; 159 patients with Child-Pugh B7 or lower were analyzed. Response (based on European Association for the Study of the Liver [EASL] and World Health Organization [WHO] criteria) was associated with survival times using the landmark, risk-of-death, and Mantel-Byar methodologies. In a subanalysis, survival times of responders were compared with those of patients with stable disease and progressive disease. Based on 6-month data, in landmark analysis, responders survived longer than nonresponders (based on EASL but not WHO criteria: P = .002 and .0694). The risk of death was also lower for responders (based on EASL but not WHO criteria: P = .0463 and .707). Landmark analysis of 12-month data showed that responders survived longer than nonresponders (P < .0001 and .004, based on EASL and WHO criteria, respectively). The risk of death was lower for responders (P = .0132 and .010, based on EASL and WHO criteria, respectively). By the Mantel-Byar method, responders had longer survival than nonresponders, based on EASL criteria (P < .0001; P = .596 with WHO criteria). In the subanalysis, responders lived longer than patients with stable disease or progressive disease. Radiographic response to LRTs predicts survival time. EASL criteria for response more consistently predicted survival times than WHO criteria. The goal of LRT should be to achieve a radiologic response, rather than to stabilize disease.
Highlights
The incidence of hepatocellular carcinoma (HCC) is increasing;[1] it has tripled in the United States from 1975-2005.2 HCC is the 6th most common malignancy worldwide and is the third most common cause of cancer-related mortality.[3,4] While surgical treatments provide the best curative outcomes, many patients present at an advanced stage
The risk of death was lower for responders
Landmark analysis of 12-month data showed that responders survived longer than nonresponders (P=
Summary
The incidence of HCC is increasing;[1] it has tripled in the United States from 1975-2005.2 HCC is the 6th most common malignancy worldwide and is the third most common cause of cancer-related mortality.[3,4] While surgical treatments (transplantation/resection) provide the best curative outcomes, many patients present at an advanced stage. Variables that can be used as surrogates of survival are of interest; these are of paramount importance in designing clinical trials. One such surrogate is radiographic tumor response. This straightforward endpoint is potentially the pinnacle upon which clinical decisions are made on an individual basis; patients and clinicians want to know if the tumor(s) has(ve) responded to treatment by exhibiting a decrease in size or demonstrating necrosis. We analyzed the association between tumor response and survival times of patients with HCC who were treated with locoregional therapies (LRTs; chemoembolization and radioembolization)
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