BACKGROUND: Pediatric dilated cardiomyopathy (DCM) is the leading indication for heart transplantation after 1 year of age. Risk factors by etiology at clinical presentation have not been determined separately for death and transplantation in population-based studies. Competing risks analysis may inform patient prioritization for transplantation listing. METHODS AND RESULTS: The Pediatric Cardiomyopathy Registry enrolled 1731 children diagnosed with DCM from 1990 to 2007. Etiologic, demographic, and echocardiographic data collected at diagnosis were analyzed with competing risks methods stratified by DCM etiology to identify predictors of death and transplantation. For idiopathic DCM (n=1192), diagnosis after 6 years of age, congestive heart failure, and lower left ventricular (LV) fractional shortening z score were independently associated with both death and transplantation equally. In contrast, increased LV end-diastolic dimension z score was associated only with transplantation, whereas lower height-for-age z score was associated only with death. For neuromuscular disease (n=139), lower LV fractional shortening was associated equally with both end points, but increased LV end-diastolic dimension was associated only with transplantation. The risks of death and transplantation were increased equally for older age at diagnosis, congestive heart failure, and increased LV end-diastolic dimension among those with myocarditis (n=272) and for congestive heart failure and decreased LV fractional shortening among those with familial DCM (n=79). CONCLUSIONS: Risk factors for death and transplantation in children varied by DCM etiology. For idiopathic DCM, increased LV end-diastolic dimension was associated with increased transplantation risk but not mortality. Conversely, short stature was significantly related to death but not transplantation. These findings may present an opportunity to improve the transplantation selection algorithm.
BACKGROUND:Pediatric dilated cardiomyopathy (DCM) is the leading indication for heart transplantation after 1 year of age. Risk factors by etiology at clinical presentation have not been determined separately for death and transplantation in population-based studies. Competing risks analysis may inform patient prioritization for transplantation listing. METHODS AND RESULTS: The Pediatric Cardiomyopathy Registry enrolled 1731 children diagnosed with DCM from 1990 to 2007. Etiologic, demographic, and echocardiographic data collected at diagnosis were analyzed with competing risks methods stratified by DCM etiology to identify predictors of death and transplantation. For idiopathic DCM (n=1192), diagnosis after 6 years of age, congestive heart failure, and lower left ventricular (LV) fractional shortening z score were independently associated with both death and transplantation equally. In contrast, increased LV end-diastolic dimension z score was associated only with transplantation, whereas lower height-for-age z score was associated only with death. For neuromuscular disease (n=139), lower LV fractional shortening was associated equally with both end points, but increased LV end-diastolic dimension was associated only with transplantation. The risks of death and transplantation were increased equally for older age at diagnosis, congestive heart failure, and increased LV end-diastolic dimension among those with myocarditis (n=272) and for congestive heart failure and decreased LV fractional shortening among those with familial DCM (n=79). CONCLUSIONS: Risk factors for death and transplantation in children varied by DCM etiology. For idiopathic DCM, increased LV end-diastolic dimension was associated with increased transplantation risk but not mortality. Conversely, short stature was significantly related to death but not transplantation. These findings may present an opportunity to improve the transplantation selection algorithm.
Authors: M A Grenier; S K Osganian; G F Cox; J A Towbin; S D Colan; P R Lurie; L A Sleeper; E J Orav; S E Lipshultz Journal: Am Heart J Date: 2000-02 Impact factor: 4.749
Authors: Piers E F Daubeney; Alan W Nugent; Patty Chondros; John B Carlin; Steven D Colan; Michael Cheung; Andrew M Davis; C W Chow; Robert G Weintraub Journal: Circulation Date: 2006-11-20 Impact factor: 29.690
Authors: Jeffrey A Towbin; April M Lowe; Steven D Colan; Lynn A Sleeper; E John Orav; Sarah Clunie; Jane Messere; Gerald F Cox; Paul R Lurie; Daphne Hsu; Charles Canter; James D Wilkinson; Steven E Lipshultz Journal: JAMA Date: 2006-10-18 Impact factor: 56.272
Authors: Steven E Lipshultz; Sarah A Vlach; Stuart R Lipsitz; Stephen E Sallan; Marcy L Schwartz; Steven D Colan Journal: Pediatrics Date: 2005-06 Impact factor: 7.124
Authors: N A Drucker; S D Colan; A B Lewis; A S Beiser; D L Wessel; M Takahashi; A L Baker; A R Perez-Atayde; J W Newburger Journal: Circulation Date: 1994-01 Impact factor: 29.690
Authors: M Giulia Gagliardi; M Bevilacqua; C Bassano; B Leonardi; R Boldrini; F Diomedi Camassei; A Fierabracci; A G Ugazio; G F Bottazzo Journal: Heart Date: 2004-10 Impact factor: 5.994
Authors: Tajinder P Singh; Lynn A Sleeper; Steven Lipshultz; Amy Cinar; Charles Canter; Steven A Webber; Daniel Bernstein; Elfriede Pahl; Jorge A Alvarez; James D Wilkinson; Jeffrey A Towbin; Steven D Colan Journal: Circ Heart Fail Date: 2009-09-28 Impact factor: 8.790
Authors: Alan S Go; Dariush Mozaffarian; Véronique L Roger; Emelia J Benjamin; Jarett D Berry; Michael J Blaha; Shifan Dai; Earl S Ford; Caroline S Fox; Sheila Franco; Heather J Fullerton; Cathleen Gillespie; Susan M Hailpern; John A Heit; Virginia J Howard; Mark D Huffman; Suzanne E Judd; Brett M Kissela; Steven J Kittner; Daniel T Lackland; Judith H Lichtman; Lynda D Lisabeth; Rachel H Mackey; David J Magid; Gregory M Marcus; Ariane Marelli; David B Matchar; Darren K McGuire; Emile R Mohler; Claudia S Moy; Michael E Mussolino; Robert W Neumar; Graham Nichol; Dilip K Pandey; Nina P Paynter; Matthew J Reeves; Paul D Sorlie; Joel Stein; Amytis Towfighi; Tanya N Turan; Salim S Virani; Nathan D Wong; Daniel Woo; Melanie B Turner Journal: Circulation Date: 2013-12-18 Impact factor: 29.690
Authors: Paolo Rusconi; James D Wilkinson; Lynn A Sleeper; Minmin Lu; Gerald F Cox; Jeffrey A Towbin; Steven D Colan; Steven A Webber; Charles E Canter; Stephanie M Ware; Daphne T Hsu; Wendy K Chung; John L Jefferies; Christina Cordero; Steven E Lipshultz Journal: Circ Heart Fail Date: 2017-02 Impact factor: 8.790
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: Emelia J Benjamin; Michael J Blaha; Stephanie E Chiuve; Mary Cushman; Sandeep R Das; Rajat Deo; Sarah D de Ferranti; James Floyd; Myriam Fornage; Cathleen Gillespie; Carmen R Isasi; Monik C Jiménez; Lori Chaffin Jordan; Suzanne E Judd; Daniel Lackland; Judith H Lichtman; Lynda Lisabeth; Simin Liu; Chris T Longenecker; Rachel H Mackey; Kunihiro Matsushita; Dariush Mozaffarian; Michael E Mussolino; Khurram Nasir; Robert W Neumar; Latha Palaniappan; Dilip K Pandey; Ravi R Thiagarajan; Mathew J Reeves; Matthew Ritchey; Carlos J Rodriguez; Gregory A Roth; Wayne D Rosamond; Comilla Sasson; Amytis Towfighi; Connie W Tsao; Melanie B Turner; Salim S Virani; Jenifer H Voeks; Joshua Z Willey; John T Wilkins; Jason Hy Wu; Heather M Alger; Sally S Wong; Paul Muntner Journal: Circulation Date: 2017-01-25 Impact factor: 29.690
Authors: Alan S Go; Dariush Mozaffarian; Véronique L Roger; Emelia J Benjamin; Jarett D Berry; William B Borden; Dawn M Bravata; Shifan Dai; Earl S Ford; Caroline S Fox; Sheila Franco; Heather J Fullerton; Cathleen Gillespie; Susan M Hailpern; John A Heit; Virginia J Howard; Mark D Huffman; Brett M Kissela; Steven J Kittner; Daniel T Lackland; Judith H Lichtman; Lynda D Lisabeth; David Magid; Gregory M Marcus; Ariane Marelli; David B Matchar; Darren K McGuire; Emile R Mohler; Claudia S Moy; Michael E Mussolino; Graham Nichol; Nina P Paynter; Pamela J Schreiner; Paul D Sorlie; Joel Stein; Tanya N Turan; Salim S Virani; Nathan D Wong; Daniel Woo; Melanie B Turner Journal: Circulation Date: 2012-12-12 Impact factor: 29.690
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