060 A randomised study of autologous bone marrow-derived stem cells in pediatric cardiomyopathy

• Published in: Archives of disease in childhood 2018-12, Vol.103 (Suppl 2), p.A25-A25
• Main Authors: Pincott, ES, Ridout, D, Brocklesby, M, McEwan, A, Muthurangu, V, Burch, M
• Format: Article
• Language: English
• Subjects: Anaphylaxis; Anesthesia; Bone imaging; Bone marrow; Bone marrow transplantation; Bone remodeling; Brain natriuretic peptide; Cardiomyopathy; Cell death; Cell therapy; Children; Dilated cardiomyopathy; Feasibility studies; Heart; Injection; Magnetic resonance imaging; Malignancy; Neuroimaging; Patients; Pediatrics; Placebos; Safety; Stem cells
• ISSN: 0003-9888; 1468-2044
• DOI: 10.1136/goshabs.60

IntroductionBone marrow mononuclear cell fraction has been used as therapy for dilated cardiomyopathy in adults. Although case series are reported, there are no randomised controlled studies in children.MethodsWe designed a randomised, crossover, controlled pilot study to determine safety and feasibility of intracoronary stem cell therapy in children. The primary safety end-point was freedom from death and transplantation or any complication that could be considered related to bone marrow injection or anaesthesia (e.g., infection, malignancy, anaphylaxis, renal deterioration). Other end-points were magnetic resonance imaging measurements and N-terminal prohormone brain natriuretic peptide. Participants with cardiomyopathy (New York Heart Association/Ross Classification II-IV) were identified, the study included 10 children (6 M; 4 F), with a mean age of 7.2 years (range, 2.2–14.1 years). Patients were crossed over at 6 months.ResultsThe original protocol was completed by 9 patients. The safety end-point was achieved in all. Ratio of the geometric means for treatment effect, adjusting for baseline, was assessed for end-diastolic and end-systolic volumes (EDV, ESV). The ratio for EDV was 0.93 (95% confidence interval 0.88–0.99, p=0.01), this indicated that EDV was on average 7% lower in patients after stem cell treatment compared with placebo. The ratio for ESV was 0.90 (95% confidence interval 0.82–1.00, p=0.05), this indicated that ESV was on average 10% lower after stem cell treatment compared with placebo. The primary efficacy end-point ejection fraction was not met.DiscussionBone marrow mononuclear cell therapy for cardiomyopathy is feasible and safe in children. Left ventricular volumes were significantly reduced 6 months after stem cell injection compared with placebo, which may reflect reverse remodelling.