OnCo
ideasIdea

A pragmatic trial of statins to prevent liver cancer in people with cirrhosis

People with cirrhosis have a high risk of liver cancer, and those who happen to take statins seem to get it less often. A proper trial would settle whether statins should be prescribed for prevention.

Large observational studies and meta-analyses associate statin use with substantially lower hepatocellular carcinoma incidence in cirrhosis and chronic hepatitis, with plausible mechanisms (reduced portal pressure, anti-fibrotic and anti-proliferative effects), and small randomised trials of simvastatin in cirrhosis show improved portal haemodynamics. No adequately powered prevention trial with cancer incidence as the endpoint has been completed. The proposal is a pragmatic randomised trial of atorvastatin or simvastatin versus placebo in compensated cirrhosis with HCC incidence as the primary outcome, run through hepatology networks and registries.

Hypothesis
Statin therapy reduces the 5-year incidence of hepatocellular carcinoma in compensated cirrhosis by at least 30% relative to placebo.
Rationale
Cirrhosis is one of the few settings where a very high-risk population is already under surveillance, making a prevention trial feasible with a few thousand patients; the drug is safe, cheap and already indicated in many of these patients for cardiovascular reasons.
What would test it
Randomised placebo-controlled trial of about 3,000 patients with compensated cirrhosis followed for five years with ultrasound surveillance; HCC incidence primary endpoint.
Maturity
early clinical
Who has to act
research
Cost to try
Medium ($1M to $50M)
Years to first evidence
7
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cancers

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Hepatocellular carcinoma

bottlenecks

2
No incentive to repurpose cheap drugsPrevention we already have is not deployed