Abstract
For more than a decade, stem cell therapy has been the focus of intensive efforts for the treatment of adult heart disease, and now has promise for treating the pediatric population. On the basis of encouraging results in the adult field, the application of stem cell-based strategies in children with congenital heart disease (CHD) opens a new therapy paradigm. To date, the safety and efficacy of stem cell-based products to promote cardiac repair and recovery in dilated cardiomyopathy and structural heart disease in infants have been primarily demonstrated in scattered clinical case reports, and supported by a few relevant pre-clinical models. Recently the TICAP trial has shown the safety and feasibility of intracoronary infusion of autologous cardiosphere-derived cells in children with hypoplastic left heart syndrome. A focus on preemptive cardiac regeneration in the pediatric setting may offer new insights as to the timing of surgery, location of cell-based delivery, and type of cell-based regeneration that could further inform acquired cardiac disease applications. Here, we review the current knowledge on the field of stem cell therapy and tissue engineering in children with CHD, and discuss the gaps and future perspectives on cell-based strategies to treat patients with CHD.
Highlights
Congenital heart disease (CHD) represents a significant burden on the family and community despite considerable advances in surgical techniques and clinical management
The main outcome is a high degree of confidence in the safety profile. These results are confirmed with numerous published meta-analyses (Table 2) [9,10,13,14,15,36]. Those studies concluded that there were no differences in major adverse events between bone marrow cell-treated and control groups, such as all-cause mortality, cardiac mortality, incidence of recurrent myocardial infarction, and in-stent thrombosis, a potential concern in patients treated with IC bone marrow derived stem cells (BMSC) infusion
When newer large randomized controlled clinical trials restricted to patients with acute myocardial infarction (AMI) were included in the latest meta-analyses, the IC infusion of bone marrow-derived mononuclear cells (BM-MNCs) did not show an improvement of cardiac function or a reduction of all-cause mortality, cardiac mortality, or recurrent AMI hospitalizations for either cardiac or neurologic complications
Summary
Congenital heart disease (CHD) represents a significant burden on the family and community despite considerable advances in surgical techniques and clinical management. Progressive late heart failure in children and young adults has become a serious problem, with an overall mortality of 7% in the United States [3]. The majority of these patients have CHD, with other causes being cardiomyopathy and myocarditis. Pressure overload in the children’s right ventricle results in a substantial increase in the production of cardiac stem cells, suggesting an adaptive response in this cohort of patients. This innate regeneration may not be sufficient to address the challenges of severe CHD [4]. This review article addresses the current knowledge in the new field of stem cell-based regenerative therapies and tissue engineering and their potential to treat children with CHD when benchmarked to the experience in the adult practice, and describes the gaps in knowledge in the field of CHD repairment
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