Cardiomyopathy and heart transplantation in children.

Cardiomyopathy is one of the most common causes of death in children with heart disease. Increasingly, dilated cardiomyopathy is recognized to be familial, and specific gene products related to the myocyte cytoskeleton and contractile proteins have been identified. Other associations with metabolic disease, dysmorphic syndromes, and neuromuscular disease are important to establish, particularly in pediatric patients, to guide therapy and patient selection for transplantation. Survival in children with dilated cardiomyopathy depends on accurate diagnosis and aggressive therapy. Patients may respond to conventional treatment for heart failure or may deteriorate, requiring mechanical support. Extracorporeal membrane oxygenation has been used effectively for mechanical support in children until improvement occurs or as a bridge to transplantation. For those who are listed, the mortality rate while waiting for a donor organ averages approximately 20%. Survival after transplantation is good, with an intermediate survival rate of approximately 70%. Late survival remains to be determined in the current cyclosporin era but may in fact be improving. However, increased organ donation or strategies to increase the size of the organ donor pool, such as xenotransplantation, are needed to significantly reduce the rate of mortality while waiting.