Outcomes of children with restrictive cardiomyopathy listed for heart transplant: a multi-institutional study.
BACKGROUND
Restrictive cardiomyopathy (RCM) in children often has a progressive nature, with a high risk of clinical deterioration and death. Heart transplantation (HTx) is a widely accepted therapy that offers long-term survival, but criteria for and outcomes after listing have not been well defined.


METHODS
A multi-institutional, prospective, event-driven data registry of 3,147 patients aged < 18 years listed for HTx from January 1993 to December 2006 was used to assess risk factors and survival of 145 listed RCM patients.


RESULTS
Mean age at listing was 8.1 years, with 44% listed as United Network of Organ Sharing status 1, 33% on inotropic support, 10% on a ventilator, and 5% on mechanical support. At 1 year, 82% of these patients survived to HTx, whereas 9% died waiting. Univariate risk factors for death while waiting included younger age (p < 0.001), ventilator dependence (p < 0.001), status 1 (p < 0.001), and inotrope usage (p < 0.001). Use of multiple support devices at listing (ventilator, extracorporeal membrane oxygenation, ventricular assist device, intraaortic balloon pump) was also an important risk factor for early phase death while waiting (relative risk; 9.01, p < 0.0001). Survival after listing was 63% at 10 years and compared favorably with survival for non-cardiomyopathy patients (p = 0.01).


CONCLUSIONS
Children with RCM awaiting HTx have a generally low waitlist mortality and reasonable overall survival. Children requiring mechanical support and infants had a significantly higher risk of death while waiting. Further study is warranted to identify factors important in determining the optimal timing of listing in children with RCM before the need for inotropic or mechanical support.
Children's Hospital of Wisconsin, Milwaukee, Wisconsin, USA. szangwill@chw.org
Steven D
SD
Zangwill
David
D
Naftel
Thomas
T
L'Ecuyer
David
D
Rosenthal
Blair
B
Robinson
James K
JK
Kirklin
Gail
G
Stendahl
Anne I
AI
Dipchand
D002313:Cardiomyopathy, Restrictive / Q000401:mortality* / Q000601:surgery; D002423:Cause of Death / Q000639:trends; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D016027:Heart Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D009864:Ontario / Q000453:epidemiology; D057234:Preoperative Period; D011379:Prognosis; D011446:Prospective Studies; D015996:Survival Rate / Q000639:trends; D013997:Time Factors; D014481:United States / Q000453:epidemiology; D014850:Waiting Lists