OBJECTIVE: Heart failure is one of the most serious complications in Duchenne muscular dystrophy (DMD). Beta-blocker medication is known to improve the prognosis of chronic heart failure of adults, but its efficacy and safety for DMD patients has not been fully assessed. Thus we conducted a multicenter open trial. METHODS:Fifty-four DMD patients participated; 41 receivedcarvedilol (BB group) and 13 did not (non BB group). All patients with an ejection fraction of less than 50% receivedangiotensin-converting enzyme inhibitor. Then, patients in BB group were started on carvedilol. The mean maintenance dose of carvedilol in BB group was 7.85+/-2.80 mg/day. Clinical signs and cardiac function were monitored regularly and statistical analysis was done. RESULTS: The survival rate free from primary endpoints (death, deterioration of heart failure and severe arrhythmia) was higher in the BB group. The survival rate free from all-cause death was also higher in the BB group, although not significantly higher. Patients with primary endpoints received lower maintenance doses of carvedilol and presented higher mean heart rates (HR) during the observation period. In the BB group, mean HR at enrollment and the reduction of mean HR were correlated with the change of ejection fraction. Although serious adverse events were rare during the introduction of carvedilol, patients with advanced cardiac dysfunction required a longer period for up-titration and frequently presented with minor complaints. CONCLUSION: The present study suggests that carvedilol is relatively safe and can prevent cardiac events even in patients with DMD.
RCT Entities:
OBJECTIVE:Heart failure is one of the most serious complications in Duchenne muscular dystrophy (DMD). Beta-blocker medication is known to improve the prognosis of chronic heart failure of adults, but its efficacy and safety for DMDpatients has not been fully assessed. Thus we conducted a multicenter open trial. METHODS: Fifty-four DMDpatients participated; 41 received carvedilol (BB group) and 13 did not (non BB group). All patients with an ejection fraction of less than 50% received angiotensin-converting enzyme inhibitor. Then, patients in BB group were started on carvedilol. The mean maintenance dose of carvedilol in BB group was 7.85+/-2.80 mg/day. Clinical signs and cardiac function were monitored regularly and statistical analysis was done. RESULTS: The survival rate free from primary endpoints (death, deterioration of heart failure and severe arrhythmia) was higher in the BB group. The survival rate free from all-cause death was also higher in the BB group, although not significantly higher. Patients with primary endpoints received lower maintenance doses of carvedilol and presented higher mean heart rates (HR) during the observation period. In the BB group, mean HR at enrollment and the reduction of mean HR were correlated with the change of ejection fraction. Although serious adverse events were rare during the introduction of carvedilol, patients with advanced cardiac dysfunction required a longer period for up-titration and frequently presented with minor complaints. CONCLUSION: The present study suggests that carvedilol is relatively safe and can prevent cardiac events even in patients with DMD.
Authors: Elizabeth M McNally; Jonathan R Kaltman; D Woodrow Benson; Charles E Canter; Linda H Cripe; Dongsheng Duan; Jonathan D Finder; William J Groh; Eric P Hoffman; Daniel P Judge; Naomi Kertesz; Kathi Kinnett; Roxanne Kirsch; Joseph M Metzger; Gail D Pearson; Jill A Rafael-Fortney; Subha V Raman; Christopher F Spurney; Shari L Targum; Kathryn R Wagner; Larry W Markham Journal: Circulation Date: 2015-05-05 Impact factor: 29.690
Authors: Rakesh K Singh; Charles E Canter; Ling Shi; Steven D Colan; Debra A Dodd; Melanie D Everitt; Daphne T Hsu; John L Jefferies; Paul F Kantor; Elfriede Pahl; Joseph W Rossano; Jeffrey A Towbin; James D Wilkinson; Steven E Lipshultz Journal: J Am Coll Cardiol Date: 2017-11-28 Impact factor: 24.094