Duchenne muscular dystrophy (DMD) is a genetic, progressive neuromuscular condition that is marked by the long-term muscle deterioration with significant implications of pulmonary and cardiac dysfunction. As such, end-stage heart failure (HF) in DMD is increasingly becoming the main cause of death in this population. The early detection of cardiomyopathy is often challenging, due to a long subclinical phase of ventricular dysfunction and difficulties in assessment of cardiovascular symptomatology in these patients who usually loose ambulation during the early adolescence. However, an early diagnosis of cardiovascular disease in patients with DMD is decisive since it allows a timely initiation of cardioprotective therapies that can mitigate HF symptoms and delay detrimental heart muscle remodelling. Echocardiography and ECG are standardly used for screening and detection of cardiovascular abnormalities in these patients, although these tools are not always adequate to detect an early, clinically asymptomatic phases of disease progression. In this regard, cardiovascular magnetic resonance (CMR) with late gadolinium enhancement is emerging as a promising method for the detection of early cardiac involvement in patients with DMD. The early detection of cardiac dysfunction allows the therapeutic institution of various classes of drugs such as corticosteroids, beta-blockers, ACE inhibitors, antimineralocorticoid diuretics and novel pharmacological and surgical solutions in the multimodal and multidisciplinary care for this group of patients. This review will focus on these challenges and available options for HF in patients with DMD.

Compared with the other conduit for RVOTR Matrix conduits showed a high incidence of failure. Our results suggest that the use of Matrix conduits for RVOTR should be considered with caution.

ObjectivesAdults with congenital heart disease (ACHD) are a growing group with end-stage heart failure. We aim to describe the outcomes of ACHD patients undergoing assessment for orthotopic heart transplant (OHT).MethodsCase notes of consecutive ACHD patients (>16 years) assessed for OHT between 2000 and 2016 at our centre were reviewed. Decision and outcome were reported as of 2017. Data were analysed in three groups: systemic left ventricle (LV), systemic right ventricle (RV) and single ventricle (SV).Results196 patients were assessed (31.8 years, 27% LV, 29% RV, 44% SV). 89 (45%) patients were listed for OHT and 67 (34%) were transplanted. 41 (21%) were unsuitable or too high risk and 36 (18%) were too well for listing. Conventional surgery was undertaken in 13 (7%) and ventricular assist device in 17 (9%) with 7 (4%) bridged to candidacy. Survival from assessment was 84.2% at 1 year and 69.7% at 5 years, with no difference between groups. Patients who were considered unsuitable for OHT (HR 11.199, p<0.001) and listed (HR 3.792, p=0.030) were more likely to die than those who were considered too well. Assessments increased over the study period.ConclusionsThe number of ACHD patients assessed for OHT is increasing. A third are transplanted with a small number receiving conventional surgery. Those who are unsuitable have a poor prognosis.