BACKGROUND: Patients with Duchenne muscular dystrophy (DMD) often have severe heart failure with a high mortality rate. Most DMD patients with cardiomyopathy became symptomatic in their early to middle teens and usually die of congestive heart failure within 2-3 years from the onset of symptoms. It has been reported that the combination of an angiotensin-converting enzyme (ACE) inhibitor and a beta-blocker has additive benefits in patients with heart failure. The aim of this study was to assess whether the combination of an ACE inhibitor and a beta-blocker is associated with long-term survival of DMD patients with left ventricular (LV) dysfunction. METHODS: We retrospectively analyzed the outcomes of 52 DMD patients who had begun treatment for heart failure with an ACE inhibitor and a beta-blocker at National Yakumo Hospital during the period from 1992 to 2005. All patients used wheelchairs in their daily lives. Patients were classified as symptomatic or asymptomatic at the initiation of treatment with these two drugs. Twelve patients who had already had apparent symptoms due to heart failure were enrolled in a treatment group. Forty patients who had no symptoms with reduced LV ejection fraction (≤ 45% in echocardiography) were enrolled in a prevention group. RESULTS: Five-year and 7-year survival rates of all patients were 93 and 84%, respectively. In the treatment group, 5-year and 7-year survival rate were 81 and 71%, respectively. Survival rate became zero at 10.9 years. In the prevention group, 5-year and 7-year survival rates were 97 and 84%, respectively, and 10-year survival rate was 72%. Nine patients in the prevention group remained event-free over 10 years. CONCLUSIONS: In this study, the combination of an ACE inhibitor and a beta-blocker had a beneficial effect on long-term survival of DMD patients with heart failure. The treatment was particularly effective for asymptomatic patients with LV dysfunction.
BACKGROUND:Patients with Duchenne muscular dystrophy (DMD) often have severe heart failure with a high mortality rate. Most DMDpatients with cardiomyopathy became symptomatic in their early to middle teens and usually die of congestive heart failure within 2-3 years from the onset of symptoms. It has been reported that the combination of an angiotensin-converting enzyme (ACE) inhibitor and a beta-blocker has additive benefits in patients with heart failure. The aim of this study was to assess whether the combination of an ACE inhibitor and a beta-blocker is associated with long-term survival of DMDpatients with left ventricular (LV) dysfunction. METHODS: We retrospectively analyzed the outcomes of 52 DMDpatients who had begun treatment for heart failure with an ACE inhibitor and a beta-blocker at National Yakumo Hospital during the period from 1992 to 2005. All patients used wheelchairs in their daily lives. Patients were classified as symptomatic or asymptomatic at the initiation of treatment with these two drugs. Twelve patients who had already had apparent symptoms due to heart failure were enrolled in a treatment group. Forty patients who had no symptoms with reduced LV ejection fraction (≤ 45% in echocardiography) were enrolled in a prevention group. RESULTS: Five-year and 7-year survival rates of all patients were 93 and 84%, respectively. In the treatment group, 5-year and 7-year survival rate were 81 and 71%, respectively. Survival rate became zero at 10.9 years. In the prevention group, 5-year and 7-year survival rates were 97 and 84%, respectively, and 10-year survival rate was 72%. Nine patients in the prevention group remained event-free over 10 years. CONCLUSIONS: In this study, the combination of an ACE inhibitor and a beta-blocker had a beneficial effect on long-term survival of DMDpatients with heart failure. The treatment was particularly effective for asymptomatic patients with LV dysfunction.
Authors: Heaseung Sophia Chung; Grace E Kim; Ronald J Holewinski; Vidya Venkatraman; Guangshuo Zhu; Djahida Bedja; David A Kass; Jennifer E Van Eyk Journal: Proc Natl Acad Sci U S A Date: 2017-11-29 Impact factor: 11.205
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: Christopher F Spurney; Francis M McCaffrey; Avital Cnaan; Lauren P Morgenroth; Sunil J Ghelani; Heather Gordish-Dressman; Adrienne Arrieta; Anne M Connolly; Timothy E Lotze; Craig M McDonald; Robert T Leshner; Paula R Clemens Journal: J Am Soc Echocardiogr Date: 2015-04-21 Impact factor: 5.251
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
Authors: Ju Lan Chun; Robert O'Brien; Min Ho Song; Blake F Wondrasch; Suzanne E Berry Journal: Stem Cells Transl Med Date: 2012-12-27 Impact factor: 6.940