Dilated cardiomyopathy is a leading cause of cardiac death in children. Approximately 30% of children die or need cardiac transplantation in the first year after establishing the diagnosis. New strategies are needed to improve the outcome in this high-risk patient population.

We present our experience in 38 patients below the age of three years, who were diagnosed with dilated cardiomyopathy and who were treated at our institution between 2006 and 2012. The treatment strategy involved institution of β-blockers and angiotensin-converting enzyme inhibitors as soon as feasible. In selected cases, pulmonary artery banding or intracoronary autologous bone marrow-derived cell therapy was performed. The median age at presentation was six months (range 1–26 months). The median follow-up age was 16 months (range 2–80 months). Kaplan–Meier analysis of survival after dilated cardiomyopathy diagnosis revealed a one-year survival of 97% and a five-year survival of 86%. The rate of freedom from death or heart transplantation was 82% at one year and 69% at five years. Surviving patients who were free of transplantation, at the follow-up at 25 months (3–80 months), showed a significant improvement in left ventricular ejection fraction (from 19±11 to 46±16%) and left ventricular end-diastolic diameter (z-score from 4.6±2.4 to 1.4±1.6). In addition, the levels of B-type natriuretic peptide improved significantly (from 3330±3840 to 171±825 pg/ml).

Our data suggest that the clinical approach described here may result in a markedly improved medium-term outcome in young children with dilated cardiomyopathy. Further studies are required to evaluate whether these approaches reduce end-points such as transplantation or death.

In spite of tremendous progress in the medical and surgical treatment of children with congenital heart disease and dilated cardiomyopathy achieved during the past few decades, for some children a heart transplant remains the only option. Clinically relevant benefits of intracoronary injection of autologous stem cells on cardiac function and remodelling have been demonstrated in adult patients with acute myocardial infarction. Experience with autologous stem cell therapy in children with severe congenital or acquired pump failure is limited to a small number of case reports.

Between 2006 and 2010, nine severely ill children were treated with intracoronary infusion of autologous bone marrow-derived mononuclear cells as part of a compassionate therapy in our centre. No procedure-related unexpected adverse events occurred. There was one patient on extracorporeal membrane oxygenation who died of haemorrhage unrelated to the procedure; three patients proceeded to heart transplantation once a donor heart became available. The other five patients showed an improvement with respect to New York Heart Association classification (greater than or equal to 1), brain natriuretic peptide serum levels, and ejection fraction.

Similar to adults, intracoronary injection of autologous bone marrow cell is technically feasible and safe for children. On the basis of our data, we propose to perform a pilot study for children with congestive heart failure, to formally assess the efficacy of intracoronary autologous bone marrow cell therapy.

To assess the feasibility of interatrial stenting for left atrial decompression in infants with hypoplastic left heart syndrome treated by a “hybrid-approach”, with bilateral surgical banding of the pulmonary arteries and percutaneous stenting of the arterial duct.

We stented the atrial septum in 5 infants aged from 21 to 77 days, making the intervention as an elective procedure in 4, but as a rescue procedure in the fifth patient, who had a restrictive foramen. The stents, comprising 2 Jo-stents of 17 millimetres hand-crimped on a balloon catheter with dimensions of 10 by 20 millimetres, and 3 premounted Genesis stents with dimensions of 10 by 19 millimetres, were placed using a 6 French long or short sheath by femoral venous access. The stents were expanded under fluoroscopic guidance to create a slightly diabolo-shaped form that fitted the septum.

The percutaneous interventions were successfully performed in all cases, producing significant improvement in clinical condition after placement. The saturations of oxygen increased from an average of 64% plus or minus 18% to 88% plus or minus 7%, (p < 0.05). During a mean follow up of 2.5 months, without any anticoagulant therapy, there were no complications related to the stenting. Surgical removal of the stents was uneventful during reconstruction of the aortic arch and creation of a bidirectional cavopulmonary connection in 4 patients, and during cardiac transplantation in one.

In the context of the hybrid approach, definitive decompression of the left atrium can be achieved by stenting the atrial septum in infants with hypoplastic left heart syndrome. Placement of the stents is safe and effective, with insertion in the form of a diabolo reducing the risk of dislocation, as well as embolisation of the stent.