Early Initiation of Enzyme Replacement Therapy in Infantile Onset Pompe Disease Improves Cardiac Outcomes: A Longitudinal Analysis.

ABSTRACT: The objective of this study is to evaluate whether early enzyme replacement therapy (ERT) initiation is associated with a lower incidence of echocardiogram abnormalities and cardiac conduction abnormalities compared to later ERT initiation. We identified a cohort of patients treated with ERT for infantile onset Pompe disease (IOPD) and evaluated their cardiac outcomes by comparing clinically collected longitudinal functional echocardiogram and electrocardiogram (EKG) data. Longitudinal mixed-effects analysis was used to compare cardiac outcomes among the cohort based on timing of ERT initiation as a continuous variable (months of age) and accounted for repeated measures in an individual patient. Time-to-event analysis and Cox regression were performed to evaluate the time to achievement of

SUBMITTER: Cohen JL 

PROVIDER: S-EPMC12828348 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature