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Upgraded heart failure therapy leads to an improved outcome of dilated cardiomyopathy in infants and toddlers

Published online by Cambridge University Press:  12 December 2014

Stefan Rupp ,
Christian Apitz ,
Leonie Tholen ,
Heiner Latus ,
Stefan H. Ostermayer ,
Dorle Schmidt ,
Jürgen Bauer  and
Dietmar Schranz
Stefan Rupp*
Affiliation:
Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany Department of Pediatrics, Klinikum Kempten, Kempten, Germany
Christian Apitz
Affiliation:
Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany
Leonie Tholen
Affiliation:
Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany
Heiner Latus
Affiliation:
Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany
Stefan H. Ostermayer
Affiliation:
Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany
Dorle Schmidt
Affiliation:
Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany
Jürgen Bauer
Affiliation:
Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany
Dietmar Schranz
Affiliation:
Pediatric Heart Center, University of Giessen and Marburg, Giessen, Germany
*
Correspondence to: S. Rupp, Pediatric Heart Center, University of Giessen and Marburg, Feulgenstrasse 12, 35392 Giessen, Germany. Tel: +49 641 994 3461; Fax: +49 641 994 3469; E-mail: stefan.rupp@paediat.med.uni-giessen.de
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Abstract

Objective

Dilated cardiomyopathy is a leading cause of cardiac death in children. Approximately 30% of children die or need cardiac transplantation in the first year after establishing the diagnosis. New strategies are needed to improve the outcome in this high-risk patient population.

Method and results

We present our experience in 38 patients below the age of three years, who were diagnosed with dilated cardiomyopathy and who were treated at our institution between 2006 and 2012. The treatment strategy involved institution of β-blockers and angiotensin-converting enzyme inhibitors as soon as feasible. In selected cases, pulmonary artery banding or intracoronary autologous bone marrow-derived cell therapy was performed. The median age at presentation was six months (range 1–26 months). The median follow-up age was 16 months (range 2–80 months). Kaplan–Meier analysis of survival after dilated cardiomyopathy diagnosis revealed a one-year survival of 97% and a five-year survival of 86%. The rate of freedom from death or heart transplantation was 82% at one year and 69% at five years. Surviving patients who were free of transplantation, at the follow-up at 25 months (3–80 months), showed a significant improvement in left ventricular ejection fraction (from 19±11 to 46±16%) and left ventricular end-diastolic diameter (z-score from 4.6±2.4 to 1.4±1.6). In addition, the levels of B-type natriuretic peptide improved significantly (from 3330±3840 to 171±825 pg/ml).

Conclusion

Our data suggest that the clinical approach described here may result in a markedly improved medium-term outcome in young children with dilated cardiomyopathy. Further studies are required to evaluate whether these approaches reduce end-points such as transplantation or death.

Keywords

Dilated cardiomyopathyheart failureβ-blockerangiotensin-converting enzyme inhibitorpulmonary artery bandingchildren
Type
Original Articles
Information
Cardiology in the Young , Volume 25 , Issue 7 , October 2015 , pp. 1300 - 1305
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Copyright
© Cambridge University Press 2014 

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References

1. Richardson, P, McKenna, W, Bristow, M, et al. Report of the 1995 World Health Organization/International Society and Federation of Cardiology Task Force on the definition and classification of cardiomyopathies. Circulation 1996; 93: 841–842.Google Scholar
2. Lipshultz, SE, Sleeper, LA, Towbin, JA, et al. The incidence of pediatric cardiomyopathy in two regions of the United States. N Engl J Med 2003; 348: 1647–1655.Google Scholar
3. Nugent, AW, Daubeney, PE, Chondros, P, et al. The epidemiology of childhood cardiomyopathy in Australia. N Engl J Med 2003; 348: 1639–1646.Google Scholar
4. Canter, CE, Shaddy, RE, Bernstein, D, et al. Indications for heart transplantation in pediatric heart disease: a scientific statement from the American Heart Association Council on Cardiovascular Disease in the Young; the Councils on Clinical Cardiology, Cardiovascular Nursing, and Cardiovascular Surgery and Anesthesia; and the Quality of Care and Outcomes Research Interdisciplinary Working Group. Circulation 2007; 115: 658–676.Google Scholar
5. Meyer, P, Filippatos, GS, Ahmed, MI, et al. Effects of right ventricular ejection fraction on outcomes in chronic systolic heart failure. Circulation 2010; 121: 252–258.Google Scholar
6. Daubeney, PE, Nugent, AW, Chondros, P, et al. Clinical features and outcomes of childhood dilated cardiomyopathy: results from a national population-based study. Circulation 2006; 114: 2671–2678.Google Scholar
7. Towbin, JA, Lowe, AM, Colan, SD, et al. Incidence, causes, and outcomes of dilated cardiomyopathy in children. JAMA 2006; 296: 1867–1876.Google Scholar
8. Arola, A, Tuominen, J, Ruuskanen, O, Jokinen, E. Idiopathic dilated cardiomyopathy in children: prognostic indicators and outcome. Pediatrics 1998; 101: 369–376.Google Scholar
9. Alexander, PM, Daubeney, PE, Nugent, AW, et al. Long-term outcomes of dilated cardiomyopathy diagnosed during childhood: results from a national population-based study of childhood cardiomyopathy. Circulation 2013; 128: 2039–2046.Google Scholar
10. Harmon, WG, Sleeper, LA, Cuniberti, L, et al. Treating children with idiopathic dilated cardiomyopathy (from the pediatric cardiomyopathy Registry). Am J Cardiol 2009; 104: 281–286.Google Scholar
11. Recla, S, Steinbrenner, B, Schranz, D. Medical therapy in dilated cardiomyopathy and pulmonary arterial banding in children. J Heart Lung Transplant 2013; 32: 1045–1046.Google Scholar
12. Schranz, D, Rupp, S, Muller, M, et al. Pulmonary artery banding in infants and young children with left ventricular dilated cardiomyopathy: a novel therapeutic strategy before heart transplantation. J Heart Lung Transplant 2013; 32: 475–481.Google Scholar
13. Rupp, S, Jux, C, Bonig, H, et al. Intracoronary bone marrow cell application for terminal heart failure in children. Cardiol Young 2012; 22: 558–563.Google Scholar
14. Bock, CT, Klingel, K, Kandolf, R. Human parvovirus B19-associated myocarditis. N Engl J Med 2010; 362: 1248–1249.Google Scholar
15. Luedde, M, Ehlermann, P, Weichenhan, D, et al. Severe familial left ventricular non-compaction cardiomyopathy due to a novel troponin T (TNNT2) mutation. Cardiovasc Res 2010; 86: 452–460.Google Scholar
16. Kantor, PF, Abraham, JR, Dipchand, AI, Benson, LN, Redington, AN. The impact of changing medical therapy on transplantation-free survival in pediatric dilated cardiomyopathy. J Am Coll Cardiol 2010; 55: 1377–1384.Google Scholar
17. Shaddy, RE, Boucek, MM, Hsu, DT, et al. Carvedilol for children and adolescents with heart failure: a randomized controlled trial. JAMA 2007; 298: 1171–1179.Google Scholar
18. Miyamoto, SD, Stauffer, BL, Nakano, S, et al. Beta-adrenergic adaptation in paediatric idiopathic dilated cardiomyopathy. Eur Heart J 2014; 35: 33–41.Google Scholar
19. Winlaw, DS, McGuirk, SP, Balmer, C, et al. Intention-to-treat analysis of pulmonary artery banding in conditions with a morphological right ventricle in the systemic circulation with a view to anatomic biventricular repair. Circulation 2005; 111: 405–411.Google Scholar
20. Assmus, B, Honold, J, Schachinger, V, et al. Transcoronary transplantation of progenitor cells after myocardial infarction. N Engl J Med 2006; 355: 1222–1232.CrossRefGoogle ScholarPubMed
21. Jeevanantham, V, Butler, M, Saad, A, Abdel-Latif, A, Zuba-Surma, EK, Dawn, B. Adult bone marrow cell therapy improves survival and induces long-term improvement in cardiac parameters: a systematic review and meta-analysis. Circulation 2012; 126: 551–568.Google Scholar
22. Fischer-Rasokat, U, Assmus, B, Seeger, FH, et al. A pilot trial to assess potential effects of selective intracoronary bone marrow-derived progenitor cell infusion in patients with nonischemic dilated cardiomyopathy: final 1-year results of the transplantation of progenitor cells and functional regeneration enhancement pilot trial in patients with nonischemic dilated cardiomyopathy. Circ Heart Fail 2009; 2: 417–423.Google Scholar