Published last month in the Journal of the American College of Cardiology (JACC), the finding by the Children’s Hospital of Michigan Pediatrician-in-Chief and Wayne State University Chair of Pediatrics Steven E. Lipshultz, M.D., and a team of national researchers associated with the Pediatric Cardiomyopathy Registry was one of several recent initiatives to call attention to pediatric cardiomyopathy. Those initiatives were among the highlights at a recent medical conference that broke new ground in understanding the causes of the ailment and improving clinical treatment for pediatric patients with this form of chronic heart disease.
The Third International Conference on Cardiomyopathy in Children, co-chaired by Dr. Lipshultz and the recently appointed associate director of the Children’s Hospital of Michigan-based Children’s Research Center of Michigan (CRCM), James D. Wilkinson, M.D., brought together more than 60 internationally recognized researchers and clinicians on pediatric cardiomyopathy. Cardiomyopathy is a chronic disease in which the heart muscle (myocardium) becomes abnormally enlarged, thickened or stiffened, causing heart dysfunction and even failure.
This complex disease results in death or heart transplant among nearly 40 percent of young cardiomyopathy patients who present with heart failure, according to the latest medical research.
“Until quite recently, many children with cardiomyopathy faced a bleak future in which death or disability was a frequent outcome,” said Dr. Lipshultz during the conference, which included researchers and clinicians from the United States, Canada, Europe and Australia. “But this unique medical meeting was able to bring together some of the world’s most accomplished cardiomyopathy investigators for two full days of sharing and insights about risk factors and effective treatments.
“For the first time ever, and after more than twenty years of work by a dedicated group of cardiomyopathy specialists, pediatric cardiomyopathy is now becoming an independent field of medical research and care – with its own national patient registry and repository, and with an ever-increasing level of research funding by the National Institutes of Health (NIH).”
Held at a conference center located only a few miles from the NIH (which sent three of its own research directors and helped fund this international conference), the quadrennial meeting was initiated by Dr. Lipshultz and the Children’s Cardiomyopathy Foundation (CCF).
The CCF, a national non-profit organization, was founded 12 years ago by CCF President Lisa Yue, a 48-year-old former marketing executive who lost two young children to the disease. Since its creation in 2002, CCF has raised more than $8 million dollars for pediatric cardiomyopathy research, education and support for families with children struggling with the disease.
“As a mother who experienced the tragedy of losing my own young children to cardiomyopathy, I have long been convinced that the key to conquering the disease is through increased research and education,” said Ms. Yue. “And for the thousands of families who have been affected by cardiomyopathy, it is encouraging to see dozens of physicians gathered in a single room committed to advancing knowledge and medical progress on the disease.
“This conference is all about hope – giving children with the disease and their families hope of eventual cures and a better future.”
In addition to Drs. Lipshultz and Wilkinson, the conference heard from Children’s Hospital of Michigan Pediatrician and Professor of Pediatrics Robert D. Ross, M.D., and Wayne State University Professor of Pediatrics and Chief of Cardiology Richard Humes, M.D, both of whom were described by Dr. Lipshultz as “world-class clinicians, educators, and researchers in children’s heart diseases.”
Dr. Lipshultz noted that 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. 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.”
While describing the new JACC study as “another compelling example of how the Children’s Hospital of Michigan is leading the way in providing high-quality pediatric care through advanced medical research,” Children’s Hospital of Michigan CEO Larry Gold said it also shows how Dr. Lipshultz’ appointment last fall as the hospital’s pediatrician-in-chief is already beginning to pay “significant dividends” in pediatric research.
“Dr. Lipshultz is an internationally recognized leader with vast experience in his field of pediatric cardiology research,” said Gold, “and he’s been the principal investigator of several landmark studies on the causes and treatment of cardiomyopathy in children.”
The study was also authored by Dr. Wilkinson, who directs the administrative coordinating center of the National Heart, Blood and Lung Institute’s (NHLB) Pediatric Cardiomyopathy Registry. Dr. Wilkinson will join the Children’s Hospital of Michigan DMC as Associate Director of the Children’s Research Center of Michigan and Wayne State University School of Medicine as a Professor of Pediatrics June 1.
Describing the crowded conference in Bethesda, Maryland, Dr. Lipshultz said “the medical issues and the research discussed here were extremely valuable – but the bottom line for all of us is finding ways to answer a crucially important question: How do we get to the best quality of life for these children with cardiomyopathy, and for their families?
“How do we minimize their pain and suffering, and how do we maximize their quality of life? By funding this conference and supporting the Pediatric Cardiomyopathy Registry studies, the Children’s Cardiomyopathy Foundation is playing a huge role in helping us find that answer. Thanks to Lisa Yue’s courage and leadership, as well as the support of other cardiomyopathy families and foundation members, and the NHLBI, the effort to help these kids is gaining ground every day.
“For a pediatric cardiologist who’s spent more than 30 years working on cardiomyopathy, this is a very exciting and hopeful time, and as we just saw at the conference in Bethesda, the prospects for real progress in eliminating and managing pediatric cardiomyopathy have never looked brighter.”
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About the Children’s Hospital of Michigan, www.childrensdmc.org
For more than 125 years, the Children’s Hospital of Michigan is the first hospital in the state dedicated exclusively to the treatment of children. With more than 40 pediatric medical and surgical specialties and services, the hospital is a leader internationally in neurology and neurosurgery, cardiology, oncology, and diagnostic services; it is ranked one of America’s best hospitals for children and sees more children than any hospital in the state. More Michigan pediatricians are trained at the Children’s Hospital of Michigan than at any other facility. Children’s Hospital of Michigan is one of eight hospitals operated by the Detroit Medical Center (DMC).
About Wayne State University, www.wayne.edu
Wayne State University is a premier urban research institution offering more than 400 academic programs through 13 schools and colleges to nearly 32,000 students. Its School of Medicine is the largest single-campus medical school in the nation with more than 1,200 medical students. In addition to undergraduate medical education, the school offers master’s degree, Ph.D. and M.D.-Ph.D. programs in 14 areas of basic science to about 400 students annually.