Clinical research and innovations in care have dramatically improved the outlook for children with many forms of heart disease. Betty Irene Moore Children’s Heart Center’s Pediatric Advanced Cardiac Therapies (PACT) program is fully committed to supporting, participating in, and leading diverse forms of research.
As a part of Stanford Medicine, our physician-scientists lead investigations into the fundamental biology, diagnoses, and care of heart failure and related conditions. We advance the understanding of heart failure and heart transplantation and discover new and improved treatments that provide options for children where sometimes none existed, decrease the risk of rejection, quicken time to transplant, improve donor matches, and increase quality of life before, during, and after transplant. If you are interested in having your child participate in a research study or clinical trial, talk with one of our doctors about opportunities.
A sampling of some of our recent and ongoing research work is listed below.
PumpKIN trial
The PumpKIN study is a national effort, funded by the National Institutes of Health, to develop a new ventricular assist device that is directed specifically at the unique needs of children with severe heart failure. Stanford investigators, led by Christopher Almond, MD, are part of the national leadership team for PumpKIN. This study is now open for enrollment at Stanford and a handful of select sites across North America.
Use of stem cells in pharmacogenomics
Under the direction of Daniel Bernstein, MD, for several decades, the Bernstein laboratory has conducted important investigations to understand cardiac function. The lab’s current work focuses on using stem cells to understand the basis for individual variation in response to cardiac medications. This will ultimately allow for personalized combinations of medications based upon these differences to maximize the benefits for patients while limiting side effects.
Panorama-HF
The Panorama-HF study is an industry-led investigation of a new medication to treat pediatric heart failure. Studies of this medication (Entresto) have shown good results in adults with heart failure. While this is encouraging, it doesn’t provide definite proof of its value in children. Panorama-HF is a large national study to answer that question. Beth Kaufman, MD, is the Stanford lead investigator for this study, which has completed enrollment for the primary phase and is now in the long-term follow-up stage.
Molecular basis of right-ventricular failure
Heart failure in children frequently involves diseases of the right ventricle, in contrast with heart failure in adults, which is more commonly seen in the left ventricle. Sushma Reddy, MD, is studying the cellular basis of right-ventricular failure in order to understand key differences and similarities between right- and left-ventricular failure. With this knowledge, we will be better able to treat this condition in affected children.
Teammate trial
This is a North American multicenter study designed to test a new combination of heart transplant immunosuppressive medications that promises to reduce the side effects associated with long-term treatment of heart transplantation patients. The study is designed and led by Christopher Almond, MD, Seth Hollander, MD, and Kevin Rady, MD, from Stanford Medicine Children’s Health and Boston Children’s Hospital. Enrollment for this study is ongoing.
