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SAT-106 Pediatric Overweight or Obesity and Early Ventricular Dilatation Are Common in Young Patients With Duchenne Muscular Dystrophy

Year: 2024

Type: article

Abstract: S. Bharill: None. Y. Liu: None. J. Vaidya: None. C. Tang: None. W.R. Thompson: None. J.L. Crane: Grant Recipient; Self; Ultragenyx, Calcylytix. Duchenne Muscular Dystrophy (DMD), an X-linked recessive disease causes progressive skeletal and cardiac muscle degeneration resulting in premature death. High dose glucocorticoid therapy slows muscle decline, but results in significant weight gain, causing overweight/obesity (OWO) and potential cardiovascular complications. To examine if OWO is associated with cardiomyopathy in DMD, we performed a retrospective analysis of 529 transthoracic echocardiograms (TTEs) on 150 pediatric and adult outpatients. Left ventricular dilatation (LVD: defined as LV End Diastolic Dimension Z-Score>1.8), ejection fraction (EF), fractional shortening (FS), strain (GLS), and myocardial work (MW: represented by Global Work Efficiency [GWE], Global Work Index [GWI], and Global Constructive Work [GCW]) were analyzed. Blood pressure (BP) and anthropometric measures (weight and height/arm span) were collected to calculate BMI and BMI z-score. Categorial variables were analyzed via Student’s t-test or Mann Whitney U. To determine if OWO or LVD were associated with worsened cardiomyopathy, mixed model analysis was performed to analyze progression of TTE outcomes after either was noted.OWO was highly prevalent, noted in 52% of patients by 10y and 72% by 20y, but decreased to 28% after >20y. Cardiac function parameters including EF, GLS, FS started in the normal range for participants <10y and became abnormal over time. LVD was often the first abnormal cardiac finding and was present in 28% of patients <10y in the absence of other cardiac abnormalities. Early LVD (prior to age 10) was not associated with OWO. OWO was associated with 6mmHg higher systolic BP (p=0.001) and increased strain- higher GLS by 1.7 units (p=0.005), but no other significant correlations with TTE outcomes. Early LVD was associated with 13.4mmHg lower systolic BP (p=0.0039) and increased MW- 1.9% lower GWE (p=0.048). LVD at any age was associated with lower BP (systolic -4.8mmHg [p=0.032], diastolic -4.1mmHg [p=0.031]) and increased MW (-171.6 GWI [p=0.0067] and -157.4 GCW [p=0.024]).While OWO did not predict worse cardiac outcomes (EF, FS, MW), a limitation of this study was lack of data on cardiac medications, which may have affected BP and other outcomes such as GLS. As OWO was associated with higher systolic BP, change in cardiac medications, a potential missing confounding variable, may have altered the course of cardiomyopathy progression. Despite this limitation, these results show that OWO and LVD are common in younger patients with DMD, and LVD is the earliest sign of cardiac involvement for some patients. Larger, longer studies with missing confounders are required to determine the relationship between OWO and cardiomyopathy and the significance of early LVD in DMD. Saturday, June 1, 2024

Source: Journal of the Endocrine Society

DOI: 10.1210/jendso/bvae163.2413

Authors: S Bharill, Y Liu, J Vaidya, C Tang, W R Thompson, J L Crane

Institution: Johns Hopkins University

Language: English

Topic: Cardiovascular Function and Risk Factors

Subfield: Cardiology and Cardiovascular Medicine

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,418