Peta M A Alexander1, Piers E F Daubeney, Alan W Nugent, Katherine J Lee, Christian Turner, Steven D Colan, Terry Robertson, Andrew M Davis, James Ramsay, Robert Justo, Gary F Sholler, Ingrid King, Robert G Weintraub. 1. From The Royal Children's Hospital, Melbourne, Victoria, Australia (P.M.A.A., A.M.D., R.G.W.); Murdoch Children's Research Institute, Melbourne, Victoria, Australia (P.M.A.A., K.J.L., A.M.D., I.K., R.G.W.); Royal Brompton Hospital and the National Heart and Lung Institute, Imperial College, London, UK (P.E.F.D.); University of Texas Southwestern Medical Center, Dallas (A.W.N.); University of Melbourne, Melbourne, Victoria, Australia (K.J.L.); Children's Hospital at Westmead, Sydney, New South Wales, Australia (C.T., G.F.S.); Boston Children's Hospital and Harvard Medical School, Boston, MA (S.D.C.); Women's and Children's Hospital, Adelaide, South Australia, Australia (T.R.); Princess Margaret Hospital, Perth, Western Australia, Australia (J.R.); and University of Queensland, Brisbane, Queensland, Australia (R.J.).
Abstract
BACKGROUND: Existing studies of childhood dilated cardiomyopathy deal mainly with early survival. This population-based study examines long-term outcomes for children with dilated cardiomyopathy. METHODS AND RESULTS: The diagnosis of dilated cardiomyopathy was based on clinical, echocardiographic, and pathological findings. The primary study end point included time to the combined outcome of death or cardiac transplantation. There were 175 patients 0 to <10 years of age at the time of diagnosis. Survival free from death or transplantation was 74% (95% confidence interval, 67-80) 1 year after diagnosis, 62% (95% confidence interval, 55-69) at 10 years, and 56% (95% confidence interval, 46-65) at 20 years. In multivariable analysis, age at diagnosis <4 weeks or >5 years, familial cardiomyopathy, and lower baseline left ventricular fractional shortening Z score were associated with increased risk of death or transplantation, as was lower left ventricular fractional shortening Z score during follow-up. At 15 years after diagnosis, echocardiographic normalization had occurred in 69% of surviving study subjects. Normalization was related to higher baseline left ventricular fractional shortening Z score, higher left ventricular fractional shortening Z score during follow-up, and greater improvement in left ventricular fractional shortening Z score. Children with lymphocytic myocarditis had better survival and a higher rate of echocardiographic normalization. At the latest follow-up, 100 of 104 of survivors (96%) were free of cardiac symptoms, and 83 (80%) were no longer receiving pharmacotherapy. CONCLUSIONS: Death or transplantation occurred in 26% of patients with childhood dilated cardiomyopathy within 1 year of diagnosis and ~1% per year thereafter. Risk factors for death or transplantation include age at diagnosis, familial cardiomyopathy, and severity of left ventricular dysfunction. The majority of surviving subjects are well and free of cardiac medication.
BACKGROUND: Existing studies of childhood dilated cardiomyopathy deal mainly with early survival. This population-based study examines long-term outcomes for children with dilated cardiomyopathy. METHODS AND RESULTS: The diagnosis of dilated cardiomyopathy was based on clinical, echocardiographic, and pathological findings. The primary study end point included time to the combined outcome of death or cardiac transplantation. There were 175 patients 0 to <10 years of age at the time of diagnosis. Survival free from death or transplantation was 74% (95% confidence interval, 67-80) 1 year after diagnosis, 62% (95% confidence interval, 55-69) at 10 years, and 56% (95% confidence interval, 46-65) at 20 years. In multivariable analysis, age at diagnosis <4 weeks or >5 years, familial cardiomyopathy, and lower baseline left ventricular fractional shortening Z score were associated with increased risk of death or transplantation, as was lower left ventricular fractional shortening Z score during follow-up. At 15 years after diagnosis, echocardiographic normalization had occurred in 69% of surviving study subjects. Normalization was related to higher baseline left ventricular fractional shortening Z score, higher left ventricular fractional shortening Z score during follow-up, and greater improvement in left ventricular fractional shortening Z score. Children with lymphocytic myocarditis had better survival and a higher rate of echocardiographic normalization. At the latest follow-up, 100 of 104 of survivors (96%) were free of cardiac symptoms, and 83 (80%) were no longer receiving pharmacotherapy. CONCLUSIONS:Death or transplantation occurred in 26% of patients with childhood dilated cardiomyopathy within 1 year of diagnosis and ~1% per year thereafter. Risk factors for death or transplantation include age at diagnosis, familial cardiomyopathy, and severity of left ventricular dysfunction. The majority of surviving subjects are well and free of cardiac medication.
Authors: Melanie D Everitt; Lynn A Sleeper; Minmin Lu; Charles E Canter; Elfriede Pahl; James D Wilkinson; Linda J Addonizio; Jeffrey A Towbin; Joseph Rossano; Rakesh K Singh; Jacqueline Lamour; Steven A Webber; Steven D Colan; Renee Margossian; Paul F Kantor; John L Jefferies; Steven E Lipshultz Journal: J Am Coll Cardiol Date: 2014-02-19 Impact factor: 24.094
Authors: Teresa M Lee; Daphne T Hsu; Paul Kantor; Jeffrey A Towbin; Stephanie M Ware; Steven D Colan; Wendy K Chung; John L Jefferies; Joseph W Rossano; Chesney D Castleberry; Linda J Addonizio; Ashwin K Lal; Jacqueline M Lamour; Erin M Miller; Philip T Thrush; Jason D Czachor; Hiedy Razoky; Ashley Hill; Steven E Lipshultz Journal: Circ Res Date: 2017-09-15 Impact factor: 17.367
Authors: Rakesh K Singh; Charles E Canter; Ling Shi; Steven D Colan; Debra A Dodd; Melanie D Everitt; Daphne T Hsu; John L Jefferies; Paul F Kantor; Elfriede Pahl; Joseph W Rossano; Jeffrey A Towbin; James D Wilkinson; Steven E Lipshultz Journal: J Am Coll Cardiol Date: 2017-11-28 Impact factor: 24.094
Authors: Gábor Mogyorósy; Enikő Felszeghy; Tamás Kovács; Andrea Berkes; László Tóth; György Balla; Ilma Korponay-Szabó Journal: Interv Med Appl Sci Date: 2014-12-22
Authors: Melanie D Everitt; James D Wilkinson; Ling Shi; Jeffrey A Towbin; Steven D Colan; Paul F Kantor; Charles E Canter; Steven A Webber; Daphne T Hsu; Elfriede Pahl; Linda J Addonizio; Debra A Dodd; John L Jefferies; Joseph W Rossano; Brian Feingold; Stephanie M Ware; Teresa M Lee; Justin Godown; Kathleen E Simpson; Lynn A Sleeper; Jason D Czachor; Hiedy Razoky; Ashley Hill; Joslyn Westphal; Kimberly M Molina; Steven E Lipshultz Journal: Prog Pediatr Cardiol Date: 2019-03-07