New research gives new insights into care of children with dilated cardiomyopathy

10th April 2017

A study looking at how well children affected by dilated cardiomyopathy do in America and Canada concludes that those with the condition in their families are more likely to have a heart transplant and it may not always be necessary.

The multi-centre study, which may influence how children are treated around the world, has just been published in Circulation Heart Failure, the journal produced by the American Heart Association.

It looked at the outcomes of children with familial dilated cardiomyopathy and dilated cardiomyopathy of an unknown cause seen at 100 children’s heart centres.

Researcher Dr Steven Lipshultz, from the Children’s Hospital of Michigan, said that children with dilated cardiomyopathy in their families are generally diagnosed at a younger age than children with dilated cardiomyopathy of an unknown cause (idiopathic).

This is because they are more likely to be screened for heart problems at an earlier age. Children with familial dilated cardiomyopathy are more likely to receive a heart transplant or intervention such as a left ventricular assist device (a heart pump) sooner due to being identified as high-risk.

He said: “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.”

The National Institutes of Health (NIH) study used data from a children’s cardiomyopathy registry set up by Dr Lipshultz and his colleagues in 1990.

According to the registry, one in 100,000 children in the US 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 may need a heart transplant or other medical interventions to survive.

Dr Lipshultz added that the second breakthrough from the study suggests that many of the children with idiopathic dilated cardiomyopathy should have a more comprehensive assessment of whether they have a genetic cause of their condition. This is because some children who are classified as having idiopathic dilated cardiomyopathy may actually have the disease in the family but not know it because of a lack of proper evaluation of the family.

 “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 counselling and treatment plans,” said Dr Lipshultz.

Luanne Thomas-Ewald, the CEO of the Children’s Hospital of Michigan, said: “With this research we are not only changing how we most appropriately treat children with heart diseases, but we are also changing the way the world thinks about this important issue.”

Cardiomyopathy UK children's support nurse Sarah Regan said: "This study shows the importance of screening for familial DCM, especially at an early age and also the importance of fully exploring the cause of all children presenting with DCM. The majority of the children transplanted at Great Ormond Street Hospital in London recently have been diagnosed with idiopathic DCM."

The study is entitled “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.”

For more details, see here.