End-stage heart failure in children or patients suffering from congenital heart disease: are new treatment options emerging?

Abstract

The development and time course of end-stage heart failure in paediatric patients suffering from cardiomyopathy and in patients suffering from repaired and unrepaired congenital heart defects are different from those of the adult population, since the myocardial damage is mainly not of an ischaemic nature. Therefore, the approach needed to treat end-stage heart failure in these patient groups is different from that in adult patients with end-stage heart failure due to coronary artery disease, cardiomyopathy or myocarditis. As in adults with acquired heart diseases, heart transplantation (HTx) and mechanical circulatory support (MCS) are the only two remaining options. However, due to issues of availability, the balance between the two options is different for children and the entire group of patients suffering from congenital heart disease (CHD). There are three major factors that most strongly impact the decision algorithm in these patients. First, due to the dramatic improvements in paediatric cardiac surgery and postoperative care, including the subsequent interventional procedures, during the past decades there have been increasing numbers of patients surviving into adulthood, such as those with univentricular hearts, who may develop end-stage heart failure after years of nearly normal quality of life [1–3]. Additionally, a significant number of adult patients who received palliative procedures as children 30–40 years ago now bear a high risk of congestive myocardial failure [4]; many of them are adults who underwent Senning and Mustard repair of transposition of the great arteries or Fontan procedures in childhood [5, 6]. Secondly, the number of paediatric HTx has stagnated while the need for donor organs continuously increases [7]. Finally, the experience with MCS lasting beyond 5 years is limited and no patients, even adults, have been supported for >8 years, whereas many children survive for >20 years after HTx. Therefore, HTx remains the gold standard in almost all children and represents the treatment of choice. However, a small proportion of children with acute myocarditis or even dilative cardiomyopathy may be better treated by MCS with subsequent weaning after remodeling of the myocardial textures and preservation of the patient’s own heart [8]. Based on these facts, the following scenarios are of special interest in the Eurotransplant area and should be discussed within the community: