Left ventricular function and response to enalapril in patients with duchenne muscular dystrophy during the second decade of life.
The role of angiotensin-converting enzyme inhibitors in the management of cardiomyopathy related to Duchenne muscular dystrophy has not been completely defined. The purposes of this study were to describe the response to enalapril and its relation to dystrophin mutation type, ventricular size, or age at the onset of left ventricular (LV) systolic dysfunction. Serial clinical and echocardiographic data from 50 patients with Duchenne muscular dystrophy (aged 10 to 20 years) were retrospectively reviewed. Twenty-seven patients (46%) developed LV systolic dysfunction (mean age 13.2 +/- 2.4 years). Ten (43%) responded to enalapril with the normalization of function. Responders and nonresponders developed LV systolic dysfunction at similar ages (p = 0.91). At the onset of LV systolic dysfunction, only 2 patients (1 responder, 1 nonresponder) had dilated left ventricles. The positive response to enalapril was sustained in 7 patients (median follow-up 23 months, range 5 to 58). No specific mutation was associated with the response to enalapril (p = 0.66) or predictive of the development of LV systolic dysfunction (p = 0.8). In conclusion, 10 of 26 patients (43%) with Duchenne muscular dystrophy responded to the use of enalapril with normalization of the shortening fraction. Age at the onset of LV systolic dysfunction and the type of mutation were not predictors of response to enalapril.
Ramaciotti
Claudio
C
Division of Cardiology, Department of Pediatrics, University of Texas, Southwestern Medical Center, Dallas, Texas, USA. claudio.ramaciotti@childrens.com
Heistein
Lisa C
LC
Coursey
Melanie
M
Lemler
Matthew S
MS
Eapen
Reenu S
RS
Iannaccone
Susan T
ST
Scott
William A
WA
D000293:Adolescent; D017668:Age of Onset; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000627:therapeutic use*; D009202:Cardiomyopathies / Q000150:complications / Q000188:drug therapy / Q000503:physiopathology; D002648:Child; D016189:Dystrophin / Q000235:genetics; D004656:Enalapril / Q000627:therapeutic use*; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000503:physiopathology*; D009154:Mutation; D018487:Ventricular Dysfunction, Left / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology; D016277:Ventricular Function, Left / Q000187:drug effects
D000806:Angiotensin-Converting Enzyme Inhibitors; D016189:Dystrophin; D004656:Enalapril