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Intracoronary bone marrow cell application for terminal heart failure in children
Stefan Rupp1, Christian Jux, Halvard Bönig
1Pediatric Heart Center, Justus-Liebig University Giessen, Germany.
Insights
Intracoronary autologous bone marrow cell therapy is safe for children with heart failure. Five of nine pediatric patients showed improved cardiac function, suggesting potential for a pilot study.
Area of Science:
- Pediatric Cardiology
- Regenerative Medicine
- Stem Cell Therapy
Background:
- Congenital heart disease and dilated cardiomyopathy in children often necessitate heart transplantation.
- Autologous stem cell therapy shows promise for cardiac repair in adults with myocardial infarction.
- Limited data exists on stem cell therapy for pediatric pump failure.
Purpose of the Study:
- To evaluate the safety and feasibility of intracoronary autologous bone marrow-derived mononuclear cell infusion in children with severe heart failure.
- To explore potential clinical benefits of this therapy in a pediatric population.
Main Methods:
- Nine severely ill children received compassionate intracoronary infusion of autologous bone marrow-derived mononuclear cells between 2006 and 2010.
- Safety and clinical outcomes, including New York Heart Association classification, brain natriuretic peptide levels, and ejection fraction, were monitored.
Main Results:
- No unexpected adverse events related to the procedure were observed.
- Five out of nine patients demonstrated significant clinical improvement.
- Improvements were noted in New York Heart Association classification, brain natriuretic peptide levels, and ejection fraction.
Conclusions:
- Intracoronary autologous bone marrow cell injection is technically feasible and safe in pediatric patients.
- The findings support conducting a pilot study to formally assess the efficacy of this therapy for pediatric congestive heart failure.
Introduction:
In spite of tremendous progress in the medical and surgical treatment of children with congenital heart disease and dilated cardiomyopathy achieved during the past few decades, for some children a heart transplant remains the only option. Clinically relevant benefits of intracoronary injection of autologous stem cells on cardiac function and remodelling have been demonstrated in adult patients with acute myocardial infarction. Experience with autologous stem cell therapy in children with severe congenital or acquired pump failure is limited to a small number of case reports.
Method And Results:
Between 2006 and 2010, nine severely ill children were treated with intracoronary infusion of autologous bone marrow-derived mononuclear cells as part of a compassionate therapy in our centre. No procedure-related unexpected adverse events occurred. There was one patient on extracorporeal membrane oxygenation who died of haemorrhage unrelated to the procedure; three patients proceeded to heart transplantation once a donor heart became available. The other five patients showed an improvement with respect to New York Heart Association classification (greater than or equal to 1), brain natriuretic peptide serum levels, and ejection fraction.
Conclusion:
Similar to adults, intracoronary injection of autologous bone marrow cell is technically feasible and safe for children. On the basis of our data, we propose to perform a pilot study for children with congestive heart failure, to formally assess the efficacy of intracoronary autologous bone marrow cell therapy.
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