News | Congenital Heart | April 04, 2017

Research Provides New Insights on Management and Clinical Outcomes for Pediatric Cardiomyopathy

Study finds different ideal management approaches for familial versus idiopathic dilated cardiomyopathy

pediatric cardiomyopathy, management, clinical outcomes, NIH study, Circulation Heart Failure, Children's Hospital of Michigan

April 4, 2017 — A multi-center study done in the United States and Canada may have global applicability for families with children afflicted with cardiomyopathy. Findings from this large National Institutes of Health (NIH) study, which have important implications in how to treat two types of dilated cardiomyopathy in children, have been published in Circulation Heart Failure, an official journal of the American Heart Association. The paper, representing findings from nearly 100 pediatric cardiology centers, is titled “Differences in Presentation and Outcomes Between Children with Familial Dilated Cardiomyopathy and Children With Idiopathic Dilated Cardiomyopathy: A Report From the Pediatric Cardiomyopathy Registry Study Group.”

Cardiomyopathy is a heart condition involving abnormalities of the muscle fibers, which contract with each heartbeat. According to the Pediatric Cardiomyopathy Registry, one in every 100,000 children in the U.S. under the age of 18 is diagnosed with cardiomyopathy. Dilated cardiomyopathy is the most common type of cardiomyopathy in infancy, childhood and adolescence. Many children newly diagnosed with dilated cardiomyopathy and heart failure do not have a good long-term prognosis and may need a heart transplant or other medical interventions in order to survive.

Children’s Hospital of Michigan pediatric cardiologist Steven E. Lipshultz, M.D., senior author and principal investigator of the study, explains that some children with dilated cardiomyopathy have other family members known to have dilated cardiomyopathy. This is called familial dilated cardiomyopathy and is mostly due to a gene mutation or set of gene mutations found in family members.

Lipshultz said that children with familial dilated cardiomyopathy are generally diagnosed at a younger age than children where their dilated cardiomyopathy is not thought to be familial, since they are more likely to be screened for heart problems at an earlier age due to other known family members affected with this condition. The children with familial dilated cardiomyopathy are more likely to receive a heart transplant or intervention such as placement of a left ventricular assist device sooner due to earlier screening and therefore being identified as high-risk.

“What this new study shows is that just because the children with familial dilated cardiomyopathy are more likely to receive a heart transplant, these heart transplants may not always be necessary since we found that these children may not die sooner or in greater numbers than children with dilated cardiomyopathy whose cause is not known to be familial. This is a critical finding since some of those children with familial dilated cardiomyopathy who received a transplant might have survived without having received a heart transplant,” he said.

Lipshultz added that the second breakthrough from this paper suggests many of the children with idiopathic dilated cardiomyopathy — meaning a cause for the child’s condition has not been identified — should have a more comprehensive assessment of whether they have a genetic cause of their condition. This is because some who are classified as idiopathic may be familial but have not been completely evaluated. This may make it a challenge for families who have a child with familial dilated cardiomyopathy since other family members who may be affected simply would not know.

“This paper suggests that genetic and echocardiographic screening of the families of all children with dilated cardiomyopathy is supported since their courses are so similar and the early identification of genetic associations or inheritance patterns may help for management, family counseling and treatment plans,” Lipshultz said.

Luanne Thomas-Ewald, CEO of the Children’s Hospital of Michigan, stated, “At the Children’s Hospital of Michigan, life is transformed by scientific advancements to achieve a better future for our patients. With this research, we are not only changing how we most appropriately treat children with heart diseases at the Children’s Hospital of Michigan, but we are also changing the way the world thinks about this important issue.”

The National Heart, Lung and Blood Institute of the NIH has funded this study as the Pediatric Cardiomyopathy Registry, which was founded by Lipshultz and his colleagues in 1990, has been funded by the NIH since 1994, and is based within the Children’s Hospital of Michigan and its Children’s Research Center of Michigan (CRCM).

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