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Title: Autologous bone marrow stem cells in paediatric heart failure
Author: Pincott, E. S.
ISNI:       0000 0004 8503 1323
Awarding Body: UCL (University College London)
Current Institution: University College London (University of London)
Date of Award: 2015
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Stem cell treatment for heart failure is a topic that generates much interest. Although heart failure is much less common in children than in adults, the social and economic implications in paediatric care are significant. The bone marrow mononuclear cell fraction has been used experimentally as therapy after myocardial infarction in adults; it appears safe and has a modest effect on ventricular function. Studies in adults with dilated cardiomyopathy have also shown positive results. Paediatric bone marrow is usually more cellular than that of adults and the patient environment possibly more receptive to potential repair and regeneration. As modest improvements are seen in adults after stem cell injections we anticipated similar injections in children to be potentially more effective. A case series has been described of stem cell therapy in children with heart failure, but no randomised studies exist. The lack of randomised studies has led to few centres considering such therapy. This randomised, crossover, placebo controlled pilot study was designed to primarily determine the safety and feasibility of stem cell intracoronary therapy in children. Secondary end points were ventricular volumes, ejection fraction and N Terminal pro B-type Natriuretic Peptide (NTproBNP). Cardiac magnetic resonance imaging was used for the assessment of cardiac volumes and function and allowed a considerable reduction in the patient numbers required to investigate the hypothesis. This is the first randomised controlled trial of the use of autologous bone marrow derived stem cells in paediatric dilated cardiomyopathy. This study demonstrated that the use of stem cells in children to address the condition of heart failure is both feasible and acceptable. No mortality or morbidity was experienced throughout the protocol, and no adverse events were reported. Left ventricular volumes were reduced at three months following stem cell injection compared with placebo, but no significant change in systolic function and NTpro-BNP were found.
Supervisor: Burch, M. ; Deanfield, J. Sponsor: Not available
Qualification Name: Thesis (Ph.D.) Qualification Level: Doctoral
EThOS ID:  DOI: Not available