69 Intracoronary Autologous Peripheral Stem Cell Therapy: A Promising Treatment for Heart Failure in Pediatric Dilated Cardiomyopathy

Abstract

<h3>Purpose</h3> Nearly one-third of children diagnosed with dilated cardiomyopathy (DCM) receive a transplant or die within the first year after diagnosis. Limited long-term outcome of heart transplantation and scarcity of young donors justify every effort to improve medical therapy and to find alternative therapeutic approaches. There is increasing evidence that cardiac stem cell transplantation induce sustained improvement in cardiac function in patients with heart failure. We describe the compassionate use of intracoronary autologous peripheral stem cell therapy in two young infants diagnosed with DCM and severe heart failure. <h3>Methods and Materials</h3> Descriptive study that analyzes the evolution of two pediatric patients diagnosed with DCM and severe heart failure, and who were treated with intracoronary infusion of autologous stem cells. Patient 1 is a 3-month male infant weighing 4 Kg. Patient 2 is a 4-month male infant weighing 5 Kg. <h3>Results</h3> At the time of admission, both patients were in poor clinical status (NYHA IV), had high levels of NT-ProBNP and severe LV dilation and systolic dysfunction (EF<30%). Both were mechanically ventilated and needed IV inotropes. Endomyocardial biopsy ruled out myocarditis. In each case, after treatment with G-CSF for 4 days, autologous stem cells were recovered from peripheral blood, implanting them via the coronary arteries afterwards. As soon as one week after the procedure, echocardiography showed a remarkable improvement in LV dilation. One month later a significant gain in their EF (>40%) was also detected and this was maintained over time. The long-term outcome was favorable. Although first patient has shown LV dilation 17 weeks after the procedure, he is still in functional class I and has adequate weight gain. <h3>Conclusions</h3> Although currently heart transplantation is the only established treatment for DCM, stem cell therapy could be an option to improve clinical status, and serve as a bridge to decision, to transplantation or, more importantly, to recovery for these very sick children.