Characterization and natural history of patients with LMNA-related dilated cardiomyopathy in the phase 3 REALM-DCM trial.

ABSTRACT:

Aims

LMNA-related dilated cardiomyopathy (DCM) is a rare disease with an incompletely defined phenotype. The phase 3 REALM-DCM trial evaluated a potential disease-modifying therapy for LMNA-related DCM but was terminated due to futility without safety concern. This study utilized pooled data from REALM-DCM to descriptively characterize the phenotype and progression of LMNA-related DCM in a contemporary cohort of patients using common heart failure (HF) measures.

Methods

REALM-DCM enrolled patients with stable LMNA-related DCM, an implanted cardioverter defibrillator or cardiac resynchronization therapy defibrillator, and New York Heart Association (NYHA) Class II/III HF symptoms.

Results

Between 2018 and 2022, 77 patients took part in REALM-DCM. The median patient age

SUBMITTER: Garcia-Pavia P 

PROVIDER: S-EPMC11631308 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature