Improvement of cardiac function by short-term enzyme replacement therapy in a murine model of cardiomyopathy associated with Hunter syndrome evaluated by serial echocardiography with speckle tracking 2-D strain analysis.

Abstract

Cardiac systolic function is significantly decreased in a proportion of patients

with Hunter syndrome. This study was performed to evaluate the change in

myocardial function associated with enzyme replacement therapy (ERT) in a mouse

model of cardiomyopathy associated with Hunter syndrome. Thirty 9-week-old

iduronate-2-sulfatase (IDS) knockout mice received either intravenous injection

of human recombinant IDS (ERT group, N=15) or saline (control group, N=15) for 5

weeks. Echocardiography was performed at baseline and after treatment.

Echocardiographic parameters of left ventricular (LV) systolic function and

2-dimensional radial and circumferential strain were assessed. At follow-up,

there was a significant increase in LV fractional shortening and radial and

circumferential strain in the ERT group only. Notable myocardial fibrosis was

observed in the control group only. In the murine model of Hunter syndrome, ERT

exerts beneficial effects on cardiac function, which can be evaluated by serial

echocardiographic evaluation including 2-dimensional strain analysis.