Effects of cardiac medications on ventricular function in patients with Duchenne muscular dystrophy-related cardiomyopathy.

INTRODUCTION/AIMS: The DMD Care Considerations Working Group Guidelines 2010 recommended treating cardiac dystrophinopathy with angiotensin-converting enzyme-inhibitor (ACEi) and beta-blocker (BB) therapy to prevent the progressive decline in left ventricular function expected from earlier, natural history studies. The aim of this research was to audit change in measures of left ventricular function over 8 years to 4 years before and 4 years after deploying an ACEi/BB combination systematically at a dedicated "cardiology-muscle" clinic. METHODS: This is an institutionally registered, retrospective, case-file-based audit of serial echocardiographic measures of left ventricular fractional shortening accumulated over the period 1995 to 2015. RESULTS: Data from 104 genetically confirmed Duchenne muscular dystrophy (DMD) patients, aged 22.2 ± 5.3 years at data censure, were included. Mean age at first detection of left ventricular dysfunction was 15.1 ± 4.2 years, but older in those on maintenance steroid therapy (16.8 ± 4.2 vs 14.5 ± 4.1 years; P = .04). Group mean fractional shortening fell by 1.5%/year over the 4 years before therapy, but this decreased to 0.9%/year over the first 4 years after starting therapy. Analysis of limited left ventricular ejection fraction measures showed similar but nonsignificant changes. Neither age at detection of left ventricular dysfunction nor fractional shortening percent at time of therapy initiation affected the beneficial response. DISCUSSION: The results support the international DMD recommendations of the time. This combination of cardiac medications helps stabilize heart function. For the best long-term effects, therapy needs to be initiated no later than on first detection left ventricular impairment.

Single-centre experience of 101 paediatric and adult Ross procedures: mid-term results.

We aimed to determine outcomes for the Ross procedure in paediatric and adult patients, with particular emphasis on survival, complication and reintervention rates. A retrospective review of 101 patients who had the Ross procedure in a congenital cardiac surgical centre serving a population of approximately 2.5 million was performed. There were 69 adults and 32 children with a mean age of 24.8 ± 13.9 years. Indications for surgery were aortic stenosis (48), regurgitation (10), mixed disease (35) and complex left outflow tract obstruction (8). The mean follow-up duration was 4.7 ± 3.7 years. The mini-inclusion technique was used to incorporate the autograft, and in all cases, pulmonary homografts were placed in the right ventricular outflow tract. Sub-aortic resection was also performed in six and Ross-Konno operations in eight patients. There were no early deaths and there was one late death secondary to endocarditis. Freedom from reintervention was 92% at 5 years and 77% at 10 years. Children were significantly more likely to require reintervention (16%, 5 of 32 versus 4%, 3 of 69, P = 0.05). The Ross procedure carries low early and mid-term mortality, and reintervention rates appear acceptable. The Ross procedure should be considered a feasible alternative to prosthetic valves in patients who require aortic valve replacement.