Practice changing RCT design and rationale: Abbreviated MRI plus AFP vs. ultrasound plus AFP for HCC surveillance in cirrhosis (PREMIUM study)

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Background Abdominal ultrasound (US) every six months with or without serum alpha-fetoprotein (AFP) is recommended for hepatocellular carcinoma (HCC) screening in patients with cirrhosis. However, high-quality evidence demonstrating that this screening strategy reduces HCC-related mortality in cirrhosis is lacking. Dynamic contrast-enhanced abbreviated MRI (DCE aMRI) protocols for HCC screening have nearly identical performance to full multiphasic MRI (the gold standard for HCC diagnosis) and can be completed in under 15 minutes. Methods PREMIUM is a multicenter RCT comparing HCC screening by US+AFP every 6 months versus DCE aMRI+AFP every 6 months for up to 8 years among cirrhosis patients. Sponsored by and executed within the Department of Veterans Affairs (VA), participant recruitment began in November 2023. Eligible participants are Veterans ages 18-75 years with cirrhosis, high HCC risk, CTP score ≤9, MELD score ≤20, and no MRI contraindications or life-threatening comorbidities. The DCE aMRI protocol consists of T1-weighted axial pre-contrast and DCE sequences (arterial, portal, and 5-minute delayed) following administration of extracellular gadolinium-based contrast agent, plus a T2-weighted sequence between portal and delayed phases. PREMIUM aims to randomize 4700 participants (2350 in each arm), who will undergo per-protocol imaging and follow-up for up to 8 years. The primary outcome is HCC-related mortality. Secondary outcomes include HCC stage at diagnosis, receipt of potentially curative HCC treatment, and all-cause mortality. The study is powered to detect at least a 35% relative reduction in HCC-related mortality in the aMRI+AFP arm versus the US+AFP arm. Discussion If PREMIUM demonstrates reduced HCC-related mortality in the aMRI+AFP arm, it could provide the necessary evidence to recommend aMRI+AFP for HCC screening in cirrhosis patients (ClinicalTrials.gov identifier: NCT05486572). Impact and Implications Specialty societies endorse ultrasound (US) for HCC screening in the cirrhosis population. However, the sensitivity and specificity of US to detect HCC at early stages is poor, and studies suggest there may be little survival benefit. Abbreviated MRI (aMRI) protocols have been developed for HCC screening that have a similarly high sensitivity and specificity to complete MRI but can be completed in a much shorter time (∼15 minutes) thus making them more suitable for screening. Abbreviated MRI protocols that include dynamic contrast enhanced images are more sensitive and specific than ultrasound and almost equally as sensitive for HCC as complete MRI. However, it is not known whether screening for HCC in patients with cirrhosis by aMRI versus ultrasound results in a reduction in HCC-related mortality. Demonstration of a reduction in HCC-related mortality in an adequately designed and powered randomized controlled trial such as PREMIUM is necessary to change the standard of care.

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