Locoregional Therapy of Hepatocellular-Cholangiocarcinoma versus Hepatocellular Carcinoma: A Propensity Score-Matched Study 

Clinical question
Do patients with unresectable hepatocellular-cholangiocarcinoma have different outcomes following locoregional therapy compared to hepatocellular carcinoma patients?

Take-away point
Patients with hepatocellular-cholangiocarcinoma have reduced performance free survival and increased distant progression following locoregional therapy compared to hepatocellular carcinoma patients.

Reference
Yu-Hui Huang, et al. Locoregional Therapy of Hepatocellular-Cholangiocarcinoma versus Hepatocellular Carcinoma: A Propensity Score-Matched Study. Journal of Vascular and Interventional Radiology. Sept, 2019: 30; 9.

Click here for abstract

Study design

Retrospective

Funding source

Self-funded or unfunded

Setting

Single-center

Figure 1. Tumor response by modified Response Evaluation Criteria in Solid Tumors following LRT of (a) the entire cohort and (b) the propensity score–matched cohort. (c) On univariate analysis, objective response rate was 30% for HCC-CC and 71% for HCC (OR 0.2, 95% CI 0.05–0.8, P = .02). (d) After propensity score matching, objective response rate was 30% for HCC-CC and 50% for HCC (OR 0.5, 95% CI 0.12–2.2, P = .3). PD = progressive disease; SD = stable disease.

Summary

Mixed hepatocellular-cholangiocarcinoma (HCC-CC) tumors are rare malignancy, comprising less than 5% of primary liver malignancies. Previously published median survival data demonstrated poor outcomes following curative resection with additional high rates of recurrence. Additionally, there has been a reported high level of recurrence following liver transplantation in this population. Currently, there is no accepted treatment paradigm for unresectable disease. Small studies have demonstrated benefit of chemoembolization in this population. The authors intended to evaluate the efficacy of local regional therapy of unresectable HCC-CC patients with propensity-scored matched patients with hepatocellular carcinoma (HCC).

10 patients with HCC-CC treated were identified. Treatments included conventional chemoembolization, radiofrequency ablation, or radioembolization with both glass and resin microspheres. Imaging was performed 1-month following treatment and at 3 month intervals and were evaluated using Response Evaluation Criteria in Solid Tumors (RECIST criteria). Within the propensity-score matched cohorts, objective response was 30% for HCC-CC and 50% for HCC. Additionally, progression free survival was shorter for the HCC-CC population, 2.4 months, compared to 6 months for HCC. HCC-CC patients also had a higher rate of distant progression (60% versus 30%).

Commentary

While small in size, this study indicates that patients with HCC-CC have worse outcomes compared to similar patients with HCC after local regional therapy. This justifies a need for dedicated treatment algorithms for HCC-CC and that adjuvant therapy may be necessary. Additionally, further investigation is needed to evaluate the efficacy of the first and second line systemic agents currently approved for HCC in the HCC-CC population.

This study has the standard limitations that come with the small sample size and retrospective nature, however given the rarity of HCC-CC, much of this is expected and difficult to avoid. The use of propensity score-matched analysis attempted to minimize confounding variables. Additionally, the study is limited due to the heterogeneity of treatment provided to the HCC-CC cohort, including a mix of resin and glass microspheres for radioembolization.

Post Author:
David M Mauro, MD
Assistant Professor
Department of Radiology, Vascular and Interventional Radiology
University of North Carolina
@DavidMauroMD