Long-Term Outcomes of Hypertrophic Cardiomyopathy Diagnosed during Childhood: Results from a National Population-Based Study

National Australian Childhood Cardiomyopathy Study

Research output: Contribution to journalArticle

10 Citations (Scopus)

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; P0.001), and lower fractional shortening z score (hazard ratio, 0.84; 95% CI, 0.74-0.95; P=0.005) during followup. 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 cardioverterdefibrillator. 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.

Original languageEnglish (US)
Pages (from-to)29-36
Number of pages8
JournalCirculation
Volume138
Issue number1
DOIs
StatePublished - Jan 1 2018

Fingerprint

Hypertrophic Cardiomyopathy
Confidence Intervals
Population
Transplantation
Cardiomyopathies
Noonan Syndrome
Left Ventricular Hypertrophy
Heart Transplantation
Survivors
Cohort Studies
Pediatrics
Equipment and Supplies
Survival

Keywords

  • Cardiovascular surgery
  • Myocardial cardiomyopathy disease
  • Pediatric and congenital heart disease

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine
  • Physiology (medical)

Cite this

Long-Term Outcomes of Hypertrophic Cardiomyopathy Diagnosed during Childhood : Results from a National Population-Based Study. / National Australian Childhood Cardiomyopathy Study.

In: Circulation, Vol. 138, No. 1, 01.01.2018, p. 29-36.

Research output: Contribution to journalArticle

National Australian Childhood Cardiomyopathy Study. / Long-Term Outcomes of Hypertrophic Cardiomyopathy Diagnosed during Childhood : Results from a National Population-Based Study. In: Circulation. 2018 ; Vol. 138, No. 1. pp. 29-36.
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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; P0.001), and lower fractional shortening z score (hazard ratio, 0.84; 95{\%} CI, 0.74-0.95; P=0.005) during followup. 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 cardioverterdefibrillator. 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.",
keywords = "Cardiovascular surgery, Myocardial cardiomyopathy disease, Pediatric and congenital heart disease",
author = "{National Australian Childhood Cardiomyopathy Study} and Alexander, {Peta M.A.} and Alan Nugent and Daubeney, {Piers E.F.} and Lee, {Katherine J.} and Sleeper, {Lynn A.} and Tibor Schuster and Christian Turner and Davis, {Andrew M.} and Chris Semsarian and Colan, {Steven D.} and Terry Robertson and James Ramsay and Robert Justo and Sholler, {Gary F.} and Ingrid King and Weintraub, {Robert G.}",
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T2 - Results from a National Population-Based Study

AU - National Australian Childhood Cardiomyopathy Study

AU - Alexander, Peta M.A.

AU - Nugent, Alan

AU - Daubeney, Piers E.F.

AU - Lee, Katherine J.

AU - Sleeper, Lynn A.

AU - Schuster, Tibor

AU - Turner, Christian

AU - Davis, Andrew M.

AU - Semsarian, Chris

AU - Colan, Steven D.

AU - Robertson, Terry

AU - Ramsay, James

AU - Justo, Robert

AU - Sholler, Gary F.

AU - King, Ingrid

AU - Weintraub, Robert G.

PY - 2018/1/1

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N2 - 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; P0.001), and lower fractional shortening z score (hazard ratio, 0.84; 95% CI, 0.74-0.95; P=0.005) during followup. 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 cardioverterdefibrillator. 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.

AB - 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; P0.001), and lower fractional shortening z score (hazard ratio, 0.84; 95% CI, 0.74-0.95; P=0.005) during followup. 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 cardioverterdefibrillator. 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.

KW - Cardiovascular surgery

KW - Myocardial cardiomyopathy disease

KW - Pediatric and congenital heart disease

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