Research Shows Hope for Normal Heart Function in Children with Fatal Heart Disease

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Newswise After two decades of arduous research, a National Institutes of Health (NIH)-funded investigator at the Childrens Hospital of Michigan (CHM) at the Detroit Medical Center (DMC) and the Wayne State University School of Medicine has published a new study showing that many children with an often fatal type of heart disease can recover normal size and function of damaged sections of their hearts.

The finding by Childrens Hospital of Michigans Pediatrician-in-Chief and Wayne State University Chair of Pediatrics Steven E. Lipshultz, M.D., F.A.A.P., F.A.H.A., clearly demonstrates that nearly one-fourth of children treated for idiopathic dilated cardiomyopathy, or DCM (also known as congestive cardiomyopathy, when these children with DCM become symptomatic by developing congestive heart failure) can be expected to fully recover normal size and function of left-ventricular (LV) heart muscle fibers within about two years of diagnosis. (LV muscle chamber size is dilated and LV function is reduced and weakened; these two abnormalities are the findings of DCM in children.)

The breakthrough from Dr. Lipshultz team of pediatric cardiology researchers, published in the April 15 issue of the authoritative Journal of the American College of Cardiology (JACC), also represents a major step forward in better understanding two of the key factors that determine survival-without-heart-transplant among children who struggle with this form of chronic heart disease, he said.

Until now, investigators have known relatively little about specific outcomes among children who are diagnosed early with DCM, said Dr. Lipshultz, who led the effort to found the nations only registry of pediatric cardiomyopathy cases (the NIH-funded North America Pediatric Cardiomyopathy Registry, or PCMR, established in 1994). But this new study is going to help change that, because it provides some revealing metrics showing that more than 22 percent of DCM-diagnosed children can be expected to regain normal heart size and function, with appropriate medical treatment and management of their condition.

Dr. Lipshultz said the recently published JACC study (http://content.onlinejacc.org/article.aspx?articleID=1833385) has the potential to provide a new sense of hope and a new opportunity for more effective treatment in the future for children diagnosed with DCM, which affects more than 100,000 children and adults worldwide (about 30,000 of them in the U.S.). Globally, DCM is the leading cause for heart transplantation in children and adolescents from 2 to 18 years of age.

The really exciting thing about this study is that it gives us a way to begin predicting which children diagnosed with DCM are most likely to survive the disease with normal heart size and function, he added. Knowing how to predict that could also be crucial in determining which children can be expected to survive without requiring heart transplants a breakthrough that could allow clinicians to reserve that extremely complex and physically demanding procedure for those patients who need it most.

The study was also authored, Dr. Lipshultz noted, by James D. Wilkinson, M.D., who directs the administrative coordinating center of the National Heart, Blood, and Lung Institute Pediatric Cardiomyopathy Registry. Dr. Wilkinson will be joining the Children's Hospital of Michigan as the Associate Director of the Children's Research Center of Michigan and Wayne State University School of Medicine as a Professor of Pediatrics as of June 1, 2014.

While describing the new JACC study as another compelling example of how the Childrens Hospital of Michigan is leading the way in providing high-quality pediatric care through advanced medical research, Childrens Hospital of Michigan CEO Larry Gold said it also shows how Dr. Lipshultz appointment last fall as the hospitals pediatrician-in-chief is already beginning to pay significant dividends in pediatric research.

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Research Shows Hope for Normal Heart Function in Children with Fatal Heart Disease