Peta M A Alexander1,2,3,4, Alan W Nugent5, Piers E F Daubeney6,7, Katherine J Lee2,8, Lynn A Sleeper3,4, Tibor Schuster2, Christian Turner9,10, Andrew M Davis11,2,8, Chris Semsarian12, Steven D Colan3,4, Terry Robertson13, James Ramsay14, Robert Justo15, Gary F Sholler9,10, Ingrid King2, Robert G Weintraub11,2,8. 1. Royal Children's Hospital, Melbourne, Australia (P.M.A.A., A.M.D., R.G.W.) peta.alexander@cardio.chboston.org. 2. Murdoch Children's Research Institute, Melbourne, Australia (P.M.A.A., K.J.L., T.S., A.M.D., I.K., R.G.W.). 3. Boston Children's Hospital, MA (P.M.A.A., L.A.S., S.D.C.). 4. Harvard Medical School, Boston, MA (P.M.A.A., L.A.S., S.D.C.). 5. University of Texas Southwestern Medical Center, Dallas (A.W.N.). 6. Royal Brompton Hospital, London, United Kingdom (P.E.F.D.). 7. National Heart and Lung Institute, Imperial College London, United Kingdom (P.E.F.D.). 8. University of Melbourne, Australia (K.J.L., A.M.D., R.G.W.). 9. Children's Hospital at Westmead, Sydney, Australia (C.T., G.F.S.). 10. University of Sydney, Australia (C.T., G.F.S.). 11. Royal Children's Hospital, Melbourne, Australia (P.M.A.A., A.M.D., R.G.W.). 12. Agnes Ginges Centre for Molecular Cardiology, Centenary Institute, University of Sydney, Australia (C.S.). 13. Women's and Children's Hospital, Adelaide, Australia (T.R.). 14. Princess Margaret Hospital, Perth, Australia (J.R.). 15. University of Queensland, Brisbane, Australia (R.J.).
Abstract
BACKGROUND: Late survival and symptomatic status of children with hypertrophic cardiomyopathy have not been well defined. We examined long-term outcomes for pediatric hypertrophic cardiomyopathy. METHODS: The National Australian Childhood Cardiomyopathy Study is a longitudinal population-based cohort study of children (0-10 years of age) diagnosed with cardiomyopathy between 1987 and 1996. The primary study end point was time to death or cardiac transplantation. RESULTS: There were 80 patients with hypertrophic cardiomyopathy, with a median age at diagnosis of 0.48 (interquartile range, 0.1, 2.5) years. Freedom from death/transplantation was 86% (95% confidence interval [CI], 77.0-92.0) 1 year after presentation, 80% (95% CI, 69.0-87.0) at 10 years, and 78% (95% CI, 67.0-86.0) at 20 years. From multivariable analyses, risk factors for death/transplantation included symmetrical left ventricular hypertrophy at the time of diagnosis (hazard ratio, 4.20; 95% CI, 1.60-11.05; P=0.004), Noonan syndrome (hazard ratio, 2.88; 95% CI, 1.02-8.08; P=0.045), higher posterior wall thickness z score (hazard ratio, 1.45; 95% CI, 1.22-1.73; P<0.001), and lower fractional shortening z score (hazard ratio, 0.84; 95% CI, 0.74-0.95; P=0.005) during follow-up. Nineteen (23%) subjects underwent left ventricular myectomy. At a median of 15.7 years of follow-up, 27 (42%) of 63 survivors were treated with β-blocker, and 13 (21%) had an implantable cardioverter-defibrillator. CONCLUSIONS: The highest risk of death or transplantation for children with hypertrophic cardiomyopathy is within 1 year after diagnosis, with low attrition rates thereafter. Many subjects receive medical, surgical, or device therapy.
BACKGROUND: Late survival and symptomatic status of children with hypertrophic cardiomyopathy have not been well defined. We examined long-term outcomes for pediatric hypertrophic cardiomyopathy. METHODS: The National Australian Childhood Cardiomyopathy Study is a longitudinal population-based cohort study of children (0-10 years of age) diagnosed with cardiomyopathy between 1987 and 1996. The primary study end point was time to death or cardiac transplantation. RESULTS: There were 80 patients with hypertrophic cardiomyopathy, with a median age at diagnosis of 0.48 (interquartile range, 0.1, 2.5) years. Freedom from death/transplantation was 86% (95% confidence interval [CI], 77.0-92.0) 1 year after presentation, 80% (95% CI, 69.0-87.0) at 10 years, and 78% (95% CI, 67.0-86.0) at 20 years. From multivariable analyses, risk factors for death/transplantation included symmetrical left ventricular hypertrophy at the time of diagnosis (hazard ratio, 4.20; 95% CI, 1.60-11.05; P=0.004), Noonan syndrome (hazard ratio, 2.88; 95% CI, 1.02-8.08; P=0.045), higher posterior wall thickness z score (hazard ratio, 1.45; 95% CI, 1.22-1.73; P<0.001), and lower fractional shortening z score (hazard ratio, 0.84; 95% CI, 0.74-0.95; P=0.005) during follow-up. Nineteen (23%) subjects underwent left ventricular myectomy. At a median of 15.7 years of follow-up, 27 (42%) of 63 survivors were treated with β-blocker, and 13 (21%) had an implantable cardioverter-defibrillator. CONCLUSIONS: The highest risk of death or transplantation for children with hypertrophic cardiomyopathy is within 1 year after diagnosis, with low attrition rates thereafter. Many subjects receive medical, surgical, or device therapy.
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