Abstract 18356: Autologous Intracoronary Stem Cell Therapy is Safe and Feasible for Children With Dilated Cardiomyopathy: A Randomised Controlled Cross-Over Pilot Study
Meta-analysis of adult stem cell therapy studies post myocardial infarction has shown moderate & sustained improvement of function. Positive results have also been seen in adult dilated cardiomyopathy (DCM) & chronic ischaemic heart failure. The place of stem cells in pediatric heart failure is unknown.
This randomised, blind, crossover placebo controlled trial sought to evaluate the potential use of stem cells in the management of pediatric heart failure & is the first of its kind within the pediatric population.
Methods & results: Ten children (mean age 7.2y, range 2.2-14.1y, 6 male) with DCM were recruited from Heart Failure clinic. Patient profiles on study entry: NYHA/Ross class 3-4, ejection fraction (EF) on MRI mean 40% (range 30-48%). On enrolment patients were randomised to receive stem cell or placebo intracoronary injection & MRI, with cross-over of intervention at 6 months & final MRI assessment at 1 year. Bone marrow processing was performed using standard techniques. The mononuclear cells, obtained within 1.5 hours of aspiration, were divided to provide 1ml for coronary injection & 1ml for phenotyping. Intracoronary injections & MRI were performed under the same anaesthetic. Mononuclear cell count mean was 51.7 x 106/ml (range 11.8-115.1 x 106/ml).
Nine patients completed the protocol, the tenth patient had Duchenne Muscular Dystrophy (DMD) & did not undergo general anaesthesia, this data was excluded from the group analysis and is presented separately. There were no withdrawals, morbidity or mortality.
Primary end point, freedom from death or transplant, was achieved in all patients. Secondary end points, indexed end diastolic volume (EDV) 6 months post cell infusion for n=9, was reduced in 7 & unchanged in 2 (P=0.01 for comparison of means), end systolic volume (ESV) decreased in 6 following cell injection. (P=0.05 for comparison of means). EDV increased in 5 patients following placebo. EF did not change significantly. EDV reduced in all patients with an injected cell count >34.2 x 106.
Conclusion: This pilot study demonstrates that intracoronary injection of autologous stem cells is safe & feasible in children. Encouraging data on EDV were generated, larger scale studies will be required to measure clinical effect.
- © 2013 by American Heart Association, Inc.