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Research & Innovation

Clinical research and less formal innovations in care have dramatically improved the outlook for children with many forms of heart disease. The PACT program is fully committed to supporting, participating in and leading diverse forms of research. A sampling of some of our recent and ongoing 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 Dr. Christopher Almond, 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 Dr. Daniel Bernstein, 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. Dr. Beth Kaufman is the Stanford lead investigator for this study, which is enrolling patients now.

Molecular basis of right-ventricular failure

Heart failure in children frequently involves diseases of the right ventricle, in contrast to heart failure in adults, which is more commonly seen in the left ventricle. Dr. Sushma Reddy 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 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 Drs. Christopher AlmondSeth Hollander and Kevin Rady from Stanford Children’s Health and Boston Children’s Hospital. Enrollment for this study will begin shortly and will continue for at least three years.