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Outcomes trials

Do CETP inhibitors have a role in treating cardiovascular disease? A review weighs harm, futility and modest benefit against emerging genomic clues (Am J Cardiovasc Drugs 2019)

Original title: Do Cholesteryl Ester Transfer Protein Inhibitors Have a Role in the Treatment of Cardiovascular Disease?

Am J Cardiovasc Drugs · · 6

Nicholls SJ, Nelson AJ

A review by two leading trialists assesses CETP inhibitors, developed on the premise that raising HDL cholesterol by blocking CETP would reduce cardiovascular events. Clinical development instead produced a spectrum of results, from evidence of harm to futility to only modest benefit, across the large-scale cardiovascular outcomes trials completed for the class. The authors survey additional insights from genomic studies suggesting potential benefit in specific clinical settings, such as particular genotypes or patient subgroups, that could still justify a role for CETP inhibition in cardiovascular risk reduction. A synthesis review assessing the state of the field rather than reporting new trial or mechanistic data.

Read the paper (DOI)PubMed

Original abstract

Cholesteryl ester transfer protein (CETP) plays an important role in lipid metabolism and has presented an attractive target for drug development, primarily resting on the hope that CETP inhibition would reduce cardiovascular events through its ability to increase levels of high-density lipoprotein cholesterol (HDL-C). However, clinical development of CETP inhibitors has proven disappointing, with a spectrum of results spanning from evidence of harm, to futility, to only modest benefit in large-scale cardiovascular outcomes trials. A number of additional insights from genomic studies have suggested potential benefits from these agents in specific clinical settings. We review the current state of CETP inhibitors as an approach to targeting cardiovascular risk.

the classgeneticsoutcomes trials

Summary written by cetpinhibition.org from the published abstract; figures as published. Page updated 18 August 2026. Methods.