Outcomes of Children With Restrictive Cardiomyopathy Listed for Heart Transplant: A Multi-institutional Study

Zangwill, Steven; Naftel, David C.; L’Ecuyer, Thomas; Rosenthal, David N.; Robinson, B.; Kirklin, James K.; Stendahl, Gail; Dipchand, Anne I.

doi:10.1016/j.healun.2009.06.028

Cited by 67 publications

(45 citation statements)

References 15 publications

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“…26 It is clear that this exceeds the natural history of typical survival for RCM patients (for whom there is no proven medical therapy that will enhance survival) and suggests that listing early in the course of the disease is generally warranted. 8 A relatively low wait-list mortality in children with RCM was observed recently by Zangwill and colleagues (in an analysis of data from the Pediatric Heart Transplant Study), 25 and both their findings, and our own, suggest that improved survival in comparison with the historical literature reflects an aggressive approach to listing and early transplantation in the current era. The Pediatric Heart Transplant Study also documented that infant age, the need for inotropic agents, mechanical ventilation, and mechanical circulatory support are important risk factors for death while waiting.…”

Section: Discussionsupporting

confidence: 66%

“…cated by the change in availability of transplantation for this condition. 24,25 Survival curves are censored at the time of transplantation, and the natural history of this condition is no longer discernable. Even more pronounced distinction from DCM and HCM is noted when one analyzes freedom from transplantation and freedom from the composite end point of death or transplantation.…”

Section: Discussionmentioning

confidence: 99%

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Outcomes of Restrictive Cardiomyopathy in Childhood and the Influence of Phenotype

et al. 2012

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(1990-2008 Nϭ3375) for cases of RCM. Cases were defined as pure when RCM was the only assigned diagnosis. Additional documentation of HCM at any time was used as the criterion for RCM/HCM phenotype. RCM accounted for 4.5% of cases of cardiomyopathy. In 101 (66%), pure RCM was diagnosed; in 51 (34%), there was a mixed phenotype. Age at diagnosis was not different between groups, but 10% of the pure RCM group was diagnosed in infancy versus 24% of the RCM/HCM group. Freedom from death was comparable between groups with 1-, 2-, and 5-year survival of RCM 82%, 80%, and 68% versus RCM/HCM 77%, 74%, and 68%. Transplant-free survival was 48%, 34%, and 22% and 65%, 53%, and 43%, respectively (Pϭ0.011). Independent risk factors at diagnosis for lower transplant-free survival were heart failure (hazard ratio 2.20, Pϭ0.005), lower fractional shortening z score (hazard ratio 1.12 per 1 SD decrease in z score, Pϭ0.014), and higher posterior wall thickness in the RCM/HCM group only (hazard ratio 1.32, PϽ0.001). Overall, outcomes were worse than for all other forms of cardiomyopathy. Conclusions-Transplant-free survival is poor for RCM in childhood. Survival is independent of phenotype; however, the RCM/HCM phenotype has significantly better transplant-free survival. Clinical Trials Registration-URL: http://www.clinicaltrials.gov. Unique Identifier: NCT00005391.

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Section: Discussionsupporting

confidence: 66%

Section: Discussionmentioning

confidence: 99%

Outcomes of Restrictive Cardiomyopathy in Childhood and the Influence of Phenotype

et al. 2012

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“…The authors reported outcomes in 1320 children with CMP (83% with dilated CMP and 17% with nondilated CMP) listed for HT during 1993-2006. 6,[8][9][10] The overall wait-list mortality was higher at 17% reflecting the earlier era (versus 11% for the composite wait-list end point in the current analysis), and posttransplant 1-year survival was 91%. Children with nondilated CMP (particularly those <1 year) and black children were reported to be at higher risk of wait-list mortality.…”

Section: Discussionmentioning

confidence: 57%

“…2 Finally, wait-list and posttransplant outcomes in children with dilated and nondilated CMP seem to have improved compared with the PHTS analyses. 6,[8][9][10] The interaction of CMP phenotype with ventilator support as a determinant of wait-list mortality is an important new finding and may be explained by examining current management options in children supported on a ventilator at listing. Although assisted ventilation can be lifesaving in children with severe heart failure, ventilator dependence is an accepted indication for VAD implantation.…”

Section: Discussionmentioning

confidence: 99%

Current Outcomes in US Children With Cardiomyopathy Listed for Heart Transplantation

Singh

Almond

Piercey

2012

Circ: Heart Failure

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Background— Previous studies have reported worse outcomes in children with nondilated cardiomyopathy (CMP) listed for heart transplant compared with children with dilated CMP. We sought to compare wait-list and posttransplant outcomes in these groups in the current era. Methods and Results— We analyzed all children <18 years of age with a diagnosis of CMP listed for heart transplant in the United States between July 2004 and December 2010. Multivariable risk factors for death on the wait-list (or becoming too sick to transplant) and posttransplant graft loss (median follow-up 2 years) were assessed using Cox models. Of the 1436 children analyzed, 1197 (83%) had dilated CMP and 239 (17%) had nondilated CMP (167 restrictive CMP, 72 hypertrophic CMP). In adjusted analysis, children with nondilated CMP were at higher risk of wait-list mortality only if they were on a ventilator support at listing (hazard ratio, 2.3; CI, 1.2–4.5). The risk was similar among children not on a ventilator support (hazard ratio, 0.6; CI, 0.3–1.1). Posttransplant 30-day and 1-year survival was 98% and 94%, respectively, in children with dilated CMP versus 95% and 89%, respectively, in children with nondilated CMP ( P =0.17, log-rank test). In adjusted analysis, the risk of posttransplant graft loss was higher in nondilated CMP (hazard ratio, 1.8; CI, 1.2–2.7) versus dilated CMP. Conclusions— The increased risk of wait-list mortality in children with nondilated CMP is limited to those on ventilator support at listing. Although the risk of graft loss is modestly higher in children with nondilated forms of CMP, their short-term transplant outcomes are good.

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“…Although the PHTS has previously published analyses of risk factors among patient subgroups, 5,6,[9][10][11][12][13][14][15][16][17][18] it has never examined risk factors in detail among the entire population of pediatric transplant recipients. Given this background, using the available data from the PHTS group, we aimed to identify independent risk factors for 1-year graft loss in pre-HTx children after stratifying by disease pathogenesis (CHD and cardiomyopathy) and to assess our ability to accurately predict graft loss using available risk factor data.…”

mentioning

confidence: 99%

Predicting Graft Loss by 1 Year in Pediatric Heart Transplantation Candidates

et al. 2015

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Background-Pediatric data on the impact of pre-heart transplantation (HTx) risk factors on early post-HTx outcomes remain inconclusive. Thus, among patients with previous congenital heart disease or cardiomyopathy, disease-specific risk models for graft loss were developed with the use pre-HTx recipient and donor characteristics. Methods and Results-Patients enrolled in the Pediatric Heart Transplant Study (PHTS) from 1996 to 2006 were stratified by pre-HTx diagnosis into cardiomyopathy and congenital heart disease cohorts. Logistic regression identified independent, pre-HTx risk factors. Risk models were constructed for 1-year post-HTx graft loss. Donor factors were added for model refinement. The models were validated with the use of patients transplanted from 2007 to 2009. Risk factors for graft loss were identified in patients with cardiomyopathy (n=896) and congenital heart disease (n=965). For cardiomyopathy, independent risk factors were earlier year of transplantation, nonwhite race, female sex, diagnosis other than dilated cardiomyopathy, higher blood urea nitrogen, and panel reactive antibody >10%. The recipient characteristic risk model had good accuracy in the validation cohort, with predicted versus actual survival of 97.5% versus 95.3% (C statistic, 0.73). For patients with congenital heart disease, independent risk factors were nonwhite race, history of Fontan, ventilator dependence, higher blood urea nitrogen, panel reactive antibody >10%, and lower body surface area. The risk model was less accurate, with 86.6% predicted versus 92.4% actual survival, in the validation cohort (C statistic, 0.63). Donor characteristics did not enhance model precision. Conclusions-Risk factors for 1-year post-HTx graft loss differ on the basis of pre-HTx cardiac diagnosis. Modeling effectively stratifies the risk of graft loss in patients with cardiomyopathy and may be an adjunctive tool in allocation policies and center performance metrics.

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Outcomes of Children With Restrictive Cardiomyopathy Listed for Heart Transplant: A Multi-institutional Study

Cited by 67 publications

References 15 publications

Outcomes of Restrictive Cardiomyopathy in Childhood and the Influence of Phenotype

Outcomes of Restrictive Cardiomyopathy in Childhood and the Influence of Phenotype

Current Outcomes in US Children With Cardiomyopathy Listed for Heart Transplantation

Predicting Graft Loss by 1 Year in Pediatric Heart Transplantation Candidates

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