[I-AEPCJS-1] Myocardial regeneration therapy for pediatric heart failure patients with single ventricle circulation
Keywords:Regeneration therapy, heart failure, single ventricle
Background: While myocardial regeneration therapy for end-stage heart failure has been reported in adult patients, there have been no coherent clinical studies or reports in pediatric population.
Methods and Results: A phase I clinical trial of regeneration therapy using autologous cardiac stem cells was conducted in 14 consecutive patients with hypoplastic left heart syndrome since January 2011 in collaboration with the Department of Cardiovascular Surgery and Pediatrics, Okayama University, and the Center for New Medical Research and Development. The safety of the therapy was confirmed in the stem cell transplant group. Right ventricular ejection fraction by echocardiography was improved with a right ventricular ejection fraction of +7.1±5.4% by echocardiography compared to that before cell transplantation. In a phase II randomized controlled comparative clinical study, 34 patients with autologous transplantation therapy, including those with other single ventricles, were treated with autologous transplantation therapy and showed significant improvement in cardiac function after 12 months. Currently, a phase III trial (validation study) has been initiated as a corporate clinical trial, and Okayama University is cooperating in the commercialization of this therapy through participation in the corporate clinical trial and clinical research.
Conclusion: In the clinical study conducted at Okayama University, autologous transplantation of cardiac stem cells improved cardiac function in single ventricle patients with heart failure, suggesting that this cardiac stem cell transplantation therapy has the potential to be a bridge or alternative treatment for patients with severe heart failure who need heart transplantation.
Methods and Results: A phase I clinical trial of regeneration therapy using autologous cardiac stem cells was conducted in 14 consecutive patients with hypoplastic left heart syndrome since January 2011 in collaboration with the Department of Cardiovascular Surgery and Pediatrics, Okayama University, and the Center for New Medical Research and Development. The safety of the therapy was confirmed in the stem cell transplant group. Right ventricular ejection fraction by echocardiography was improved with a right ventricular ejection fraction of +7.1±5.4% by echocardiography compared to that before cell transplantation. In a phase II randomized controlled comparative clinical study, 34 patients with autologous transplantation therapy, including those with other single ventricles, were treated with autologous transplantation therapy and showed significant improvement in cardiac function after 12 months. Currently, a phase III trial (validation study) has been initiated as a corporate clinical trial, and Okayama University is cooperating in the commercialization of this therapy through participation in the corporate clinical trial and clinical research.
Conclusion: In the clinical study conducted at Okayama University, autologous transplantation of cardiac stem cells improved cardiac function in single ventricle patients with heart failure, suggesting that this cardiac stem cell transplantation therapy has the potential to be a bridge or alternative treatment for patients with severe heart failure who need heart transplantation.