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Stem cell therapy for CHD: towards translation*

Published online by Cambridge University Press:  17 September 2015

Brody Wehman ,
Osama T. Siddiqui ,
Rachana Mishra ,
Sudhish Sharma  and
Sunjay Kaushal
Brody Wehman
Affiliation:
Division of Cardiac Surgery, University of Maryland School of Medicine, Baltimore, Maryland, United States of America
Osama T. Siddiqui
Affiliation:
Division of Cardiac Surgery, University of Maryland School of Medicine, Baltimore, Maryland, United States of America
Rachana Mishra
Affiliation:
Division of Cardiac Surgery, University of Maryland School of Medicine, Baltimore, Maryland, United States of America
Sudhish Sharma
Affiliation:
Division of Cardiac Surgery, University of Maryland School of Medicine, Baltimore, Maryland, United States of America
Sunjay Kaushal*
Affiliation:
Division of Cardiac Surgery, University of Maryland School of Medicine, Baltimore, Maryland, United States of America
*
Correspondence to: S. Kaushal, MD, PhD, Division of Cardiac Surgery, University of Maryland Medical Center, 110S. Paca St., 7th Floor, Baltimore, MD 21201, United States of America. Tel: +410 328 5842; Fax: +410 328 2750; E-mail: skaushal@smail.umaryland.edu
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Abstract

Stem cell therapy has the optimistic goal of regenerating the myocardium as defined by re-growth of lost or destroyed myocardium. As applied to patients with heart failure, many confuse or limit the regenerative definition to just improving myocardial function and/or decreasing myocardial scar formation, which may not be the most important clinical outcome to achieve in this promising field of molecular medicine. Many different stem cell-based therapies have been tested and have demonstrated a safe and feasible profile in adult patients with heart failure, but with varied efficacious end points reported. Although not achieved as of yet, the encompassing goal to regenerate the heart is still believed to be within reach using these cell-based therapies in adult patients with heart failure, as the first-generation therapies are now being tested in different phases of clinical trials. Similar efforts to foster the translation of stem cell therapy to children with heart failure have, however, been limited. In this review, we aim to summarise the findings from pre-clinical models and clinical experiences to date that have focussed on the evaluation of stem cell therapy in children with heart failure. Finally, we present methodological considerations pertinent to the design of a stem cell-based trial for children with heart failure, as they represent a population of patients with very different sets of issues when compared with adult patients. As has been taught by many learned clinicians, children are not small adults!

Keywords

CHDstem cellhypoplastic left heart syndromesingle ventricle
Type
Original Articles
Information
Cardiology in the Young , Volume 25 , Supplement S2: Special Supplement of Cardiology in the Young: Proceedings of the 2015 International Paediatric Heart Failure Summit of Johns Hopkins All Children's Heart Institute , August 2015 , pp. 58 - 66
Copyright
© Cambridge University Press 2015 

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Footnotes

*

Presented at Johns Hopkins All Children’s Heart Institute, International Pediatric Heart Failure Summit, Saint Petersburg, Florida, United States of America, 4–5 February, 2015.

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