ABSTRACT
The accepted treatment strategy for hepatocellular carcinoma (HCC) is supported by
randomized controlled trials (RCTs), meta-analysis, and large cohort studies. For
instance, the Milan criteria applied for indicating liver transplantation have been
validated by several cohort studies including more than 1000 patients. Regarding medical
treatments, approximately 80 RCTs have been published so far in HCC. These studies
provide the evidence to support chemoembolization as the treatment for patients at
intermediate stage (meta-analysis of 6 RCTs) and show the lack of benefit of tamoxifen
assessed in 12 RCTs including more than 1500 patients. In this scenario, what is the
evidence to advocate for the expansion of HCC criteria through down-staging prior
to liver transplantation? Such an approach has never been tested through RCTs or even
well-designed cohort studies including enough patients and adequate follow-up. Only
a few small studies with heterogeneous target populations and treatments applied are
available. The results of these studies are inconsistent and do not provide compelling
evidence to accept down-staging as a standard of care.
KEYWORDS
Hepatocellular carcinoma - liver transplantation - waiting list - down-staging - dropout
- expansion of criteria
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Josep M LlovetM.D.
Division of Liver Diseases, Mount Sinai School of Medicine
1425 Madison Avenue, 11F-70, Box 1104, New York, NY 10029