Year: 2006
Type: article
Study design: Observational Study
Abstract: INTRODUCTION: Duchenne muscular dystrophy (DMD) is characterized by a cardiomyopathy, or a predictable decline in cardiac function with age, that contributes to early death.Although steroids are the only pharmacologic therapy, there is limited published work documenting its impact on cardiomyopathy.Thus, the primary objective of this work is to dissect the causal relation of benefit from steroid treatment on the development of ventricular dysfunction in DMD.METHODS: With IRB approval, a clinical database was queried for DMD cases undergoing cardiac evaluation.Basic medical data and serial echocardiographic measures were obtained for each identified case meeting inclusion criteria.RESULTS: Data was collected from untreated (n = 23) and steroid treated (n = 14) cases from 7.5 + 0.8 years to 12 + 0.65 years.Kaplan-Meier freedom from ventricular dysfunction was 93% for steroid treated cases vs. 53% for untreated at 1500 days.Treatment with steroids was protective against ventricular dysfunction (Hazard ratio 0.16 95% CI 0.04, 0.70).CONCLUSIONS: We demonstrate here that steroid treatment, begun prior to ventricular dysfunction has a dramatic beneficial impact on the anticipated development of ventricular dysfunction.
Author: Larry W. Markham
Institution: University of Cincinnati
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W2600054707