Novel therapies in childhood heart failure: today and tomorrow.

Heart failure is an important cause of morbidity and mortality in individuals of all ages. The many-faceted nature of the clinical heart failure syndrome has historically frustrated attempts to develop an overarching explanative theory. However, much useful information has been gained by basic and clinical investigation, even though a comprehensive understanding of heart failure has been elusive. Heart failure is a growing problem, in both adult and pediatric populations, for which standard medical therapy, as of 2010, can have positive effects, but these are usually limited and progressively diminish with time in most patients. If we want curative or near-curative therapy that will return patients to a normal state of health at a feasible cost, much better diagnostic and therapeutic technologies need to be developed. This review addresses the vexing group of heart failure etiologies that include cardiomyopathies and other ventricular dysfunctions of various types, for which current therapy is only modestly effective. Although there are many unique aspects to heart failure in patients with pediatric and congenital heart disease, many of the innovative approaches that are being developed for the care of adults with heart failure will be applicable to heart failure in childhood.