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Left ventricular dysfunction in Duchenne muscular dystrophy

Published online by Cambridge University Press:  22 January 2020

Katherine A. James Open the ORCID record for Katherine A. James [Opens in a new window] ,
Jane Gralla ,
Leslie A. Ridall ,
ThuyQuynh N. Do ,
Angela S. Czaja ,
Peter M. Mourani ,
Emma Ciafaloni ,
Christopher Cunniff ,
Jennifer Donnelly  and
Joyce Oleszek
...Show all authors
Katherine A. James*
Affiliation:
Colorado School of Public Health, University of Colorado, Aurora, CO, USA
Jane Gralla
Affiliation:
Department of Pediatrics, University of Colorado, Aurora, CO, USA
Leslie A. Ridall
Affiliation:
Children’s Hospital Colorado, Aurora, CO, USA
ThuyQuynh N. Do
Affiliation:
National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA
Angela S. Czaja
Affiliation:
Children’s Hospital Colorado, Aurora, CO, USA
Peter M. Mourani
Affiliation:
Children’s Hospital Colorado, Aurora, CO, USA
Emma Ciafaloni
Affiliation:
Department of Neurology, University of Rochester, Rochester, NY, USA
Christopher Cunniff
Affiliation:
Department of Pediatrics, Weill Cornell Medical College, New York, NY, USA
Jennifer Donnelly
Affiliation:
Children’s Hospital Colorado, Aurora, CO, USA
Joyce Oleszek
Affiliation:
Children’s Hospital Colorado, Aurora, CO, USA
Shree Pandya
Affiliation:
Department of Neurology, University of Rochester, Rochester, NY, USA
Elinora Price
Affiliation:
Department of Pediatrics, Weill Cornell Medical College, New York, NY, USA
Michele L. Yang
Affiliation:
Children’s Hospital Colorado, Aurora, CO, USA
Scott R. Auerbach
Affiliation:
Children’s Hospital Colorado, Aurora, CO, USA
*
Author for correspondence: K. A. James, Colorado School of Public Health, University of Colorado, Anschutz Medical Campus, 13001 E 17th PL MS B119 Bldg 500 3rd FLR, Aurora, CO 80045, USA. Tel: 303-724-8169; Fax: 303.724.4620; E-mail: [email protected]
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Abstract

Background:

Duchenne muscular dystrophy is associated with progressive cardiorespiratory failure, including left ventricular dysfunction.

Methods and Results:

Males with probable or definite diagnosis of Duchenne muscular dystrophy, diagnosed between 1 January, 1982 and 31 December, 2011, were identified from the Muscular Dystrophy Surveillance Tracking and Research Network database. Two non-mutually exclusive groups were created: patients with ≥2 echocardiograms and non-invasive positive pressure ventilation-compliant patients with ≥1 recorded ejection fraction. Quantitative left ventricular dysfunction was defined as an ejection fraction <55%. Qualitative dysfunction was defined as mild, moderate, or severe. Progression of quantitative left ventricular dysfunction was modelled as a continuous time-varying outcome. Change in qualitative left ventricle function was assessed by the percentage of patients within each category at each age. Forty-one percent (n = 403) had ≥2 ejection fractions containing 998 qualitative assessments with a mean age at first echo of 10.8 ± 4.6 years, with an average first ejection fraction of 63.1 ± 12.6%. Mean age at first echo with an ejection fraction <55 was 15.2 ± 3.9 years. Thirty-five percent (140/403) were non-invasive positive pressure ventilation-compliant and had ejection fraction information. The estimated rate of decline in ejection fraction from first ejection fraction was 1.6% per year and initiation of non-invasive positive pressure ventilation did not change this rate.

Conclusions:

In our cohort, we observed that left ventricle function in patients with Duchenne muscular dystrophy declined over time, independent of non-invasive positive pressure ventilation use. Future studies are needed to examine the impact of respiratory support on cardiac function.

Keywords

Duchenne muscular dystrophynon-invasive positive pressure ventilationleft ventricular dysfunction
Type
Original Article
Information
Cardiology in the Young , Volume 30 , Issue 2 , February 2020 , pp. 171 - 176
Copyright
© Cambridge University Press 2020

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