Key result
Eplerenone is evaluated to attenuate disease onset and progression in asymptomatic PLN p.Arg14del carriers.
Why the study?
Cardiac fibrotic remodelling occurs early in inherited PLN p.Arg14del cardiomyopathy, and eplerenone was deemed a treatment candidate due to its antifibrotic properties and beneficial effects on ventricular remodelling.
Does eplerenone attenuate disease onset and progression in asymptomatic phospholamban p.Arg14del carriers?
RCT
Yes
Does eplerenone attenuate disease onset and progression in asymptomatic phospholamban p.Arg14del carriers?
Provides first RCT of eplerenone in asymptomatic PLN p.Arg14del carriers; extends evaluation to preclinical genetic cardiomyopathy prevention.
INTRODUCTION (PLN; p.Arg14del) cardiomyopathy is an inherited disease caused by the pathogenic p.Arg14del variant in the PLN gene. Clinically, it is characterized by malignant ventricular arrhythmias and progressive heart failure.1,2 Cardiac fibrotic tissue remodelling occurs early on in PLN p.Arg14del carriers.3,4 Eplerenone was deemed a treatment candidate because of its beneficial effects on ventricular remodelling and antifibrotic properties.5,6 We conducted the multicentre randomized trial ‘intervention in PHOspholamban RElated CArdiomyopathy STudy’ (i-PHORECAST) to assess whether treatment with eplerenone of asymptomatic PLN p.Arg14del carriers attenuates disease onset and progression.
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Brouwer et al. (2023) conducted an RCT in Phospholamban (PLN; p.Arg14del) cardiomyopathy. Eplerenone was evaluated on Disease onset and progression. The i-PHORECAST multicenter randomized trial was designed to evaluate whether eplerenone attenuates disease onset and progression in asymptomatic PLN p.Arg14del carriers.
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