This study summary is an excerpt from the book 2 Minute Medicine’s The Classics in Medicine: Summaries of the Landmark Trials
1. In patients with hepatocellular carcinoma (HCC) not eligible for surgical resection, the use of radiofrequency thermal ablation (RFA) demonstrated significantly higher local recurrence-free survival compared to percutaneous ethanol injection (PEI).
Original Date of Publication: July 2003
Study Rundown: HCC is the second-leading cause of cancer mortality worldwide and often occurs in the setting of cirrhosis and chronic liver disease. Curative treatment can be obtained with surgical resection or liver transplantation; however, a large proportion of patients are poor candidates for curative treatment due to poor underlying liver function. Both PEI and RFA are alternative modalities that induce local tumor necrosis in patients with HCC with underlying cirrhosis who are not eligible for surgical intervention. The purpose of this randomized controlled trial was to compare the effectiveness of PEI and RFA in patients with HCC. The trial randomized over 100 patients with HCC ineligible for surgical resection or liver transplantation to either RFA or PEI, both performed with ultrasound guidance. Patients were excluded if they had advanced liver disease (Child Pugh class C) or evidence of extra-hepatic disease or vascular invasion. At the conclusion of the trial, there was a significantly higher rate of local recurrence-free survival at 1- and 2 years in the RFA group compared to PEI. Additionally, there was a trend towards the RFA group for improved overall survival, which did not reach statistical significance. The mean number of treatment sessions was significantly lower in the RFA group with no significant increase in the complication rate compared to PEI. The results of this study demonstrate a superiority of RFA compared to PEI and support the use of RFA as the optimal alternative modality in HCC patients ineligible for curative surgical intervention. However, this study does not provide data for survival beyond two-year follow-up; as such, the long-term benefit of RFA over PEI is unknown.
Click to read the study in Radiology
In-Depth [randomized controlled trial]: This was an open-labeled, multi-center randomized controlled trial analyzing the effectiveness of RFA to PEI in patients with HCC not eligible for curative surgical interventions. The inclusion criteria included all adult patients with cirrhosis and a concurrent single HCC < 5 cm in diameter or as many as 3 HCCs with a maximum dimension of < 3 cm in diameter. Patients must be ineligible for surgical intervention or liver transplantation. Key exclusion criteria included presence of extrahepatic disease or evidence of tumor vascular invasion. Patients were also excluded if the HCCs were located less than 1 cm away from the hepatic hilum or gallbladder. The primary outcome was overall survival with a secondary endpoint of local recurrence-free survival rate. Each RFA or PEI procedure was standardized and supervised by a trial co-investigator. Follow-up for evaluation of local recurrence was done by serum α1-fetoprotein and abdominal ultrasound at three-month intervals and dual phase spiral computed tomography at 6 month intervals. Overall, 50 patients were randomized to the PEI group and 52 patients were randomized to the RFA group. There were no statistically significant differences in baseline characteristics in each treatment group, with the exception of patient age and albumin concentration. Patients were followed up for mean period of 22.9 months and 22.4 months for the RFA and PEI group, respectively. At the conclusion of the trial, the 1- and 2 year local recurrence survival rate was significantly higher in the RFA group compared to the PEI group (RR: 0.17; 95%CI 0.06-0.51; p = 0.002). There was a trend towards increased 1- and 2 year overall survival in the RFA group compared to PEI, which did not reach statistical significance (RR: 0.20; 95%CI 0.02-1.69; p = 0.138). Patients in the PEI group had an average of 5.4 (+/- 1.6) PEI sessions while the RFA group had an average of 1.1 (+/- 0.5) sessions. There was no significant difference in the frequency of adverse events such as post-procedural pain and fever. There were no procedure-related deaths, hemorrhage, or infection noted in either treatment group.
Lencioni RA, Allgaier H-P, Cioni D, Olschewski M, Deibert P, Crocetti L, et al. Small Hepatocellular Carcinoma in Cirrhosis: Randomized Comparison of Radio-frequency Thermal Ablation versus Percutaneous Ethanol Injection. Radiology. 2003 Jul 1;228(1):235–40.
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