Clinical cardiac regenerative studies in children

Abstract

Although the incidence of pediatric heart failure is low, the mortality is relatively high, with severe clinical symptoms requiring repeated hospitalization or intensive care treatment in the surviving patients. Cardiac biopsy specimens have revealed a higher number of resident human cardiac progenitor cells, with greater proliferation and differentiation capacity, in the neonatal period as compared with adults, demonstrating the regeneration potential of the young heart, with rising interest in cardiac regeneration therapy in critically ill pediatric patients. We review here the available literature data, searching the MEDLINE, Google Scholar and EMBASE database for completed, and www.clinicaltrials.gov homepage for ongoing studies involving pediatric cardiac regeneration reports. Because of difficulties conducting randomized blinded clinical trials in pediatric patients, mostly case reports or cohort studies with a limited number of individuals have been published in the field of pediatric regenerative cardiology. The majority of pediatric autologous cell transplantations into the cardiac tissue have been performed in critically ill children with severe or terminal heart failure. Congenital heart disease, myocarditis, and idiopathic hypertrophic or dilated cardiomyopathy leading to congestive heart failure are some possible areas of interest for pediatric cardiac regeneration therapy. Autologous bone marrow mononuclear cells, progenitor cells, or cardiospheres have been applied either intracoronary or percutaneously intramyocardially in severely ill children, leading to a reported clinical benefit of cell-based cardiac therapies. In conclusion, compassionate use of autologous stem cell administration has led to at least short-term improvement in heart function and clinical stability in the majority of the critically ill pediatric patients.

Keywords: Congenital heart disease; Heart failure; Cardiac regeneration; Cell-based therapy; Hospitalization; Children

Core tip: This review summarizes the available literature data involving pediatric cardiac regeneration reports. Due to lack of randomized blinded clinical trials in pediatric cardiology patients, mostly case reports with limited number of individuals have been published in the pediatric regenerative cardiology. The majority of pediatric autologous cell transplantations into the cardiac tissue have been performed in children with severe or terminal heart failure, and led to the conclusion, that compassionate use of autologous stem cell administration may lead to at least short-term improvement in heart function and clinical stability in the majority of the critically ill pediatric patients.