Children’s National leads patient-centered study of adult congenital heart disease

Investigators at Children’s National Hospital and the Louisiana Public Health Institute are leading a $4.9 million research effort to study how gaps in health care affect the health and well-being of adults with congenital heart disease (CHD), supported by the Patient-Centered Outcomes Research Institute (PCORI).

The research is led by Anitha John, M.D., Ph.D., medical director of the Washington Adult Congenital Heart Program at Children’s National Hospital and Thomas Carton, Ph.D., chief data officer at the Louisiana Public Health Institute, as well as two patient co-investigators, Ruth Phillippi and Scott Leezer. The study leads also include representatives from the Adult Congenital Heart Association and Anu Agarwal, M.D., who represents the University of California – San Francisco (UCSF).

“With the increasing number of adult patients with CHD, it is important for us to understand how current recommended practices influence patient outcomes,” says Dr. John. “This project will guide us on how to best care for our patients, not just through childhood, but across their entire lifespans. Most importantly, this project will involve direct outreach to patients, incorporating patient reported outcomes as a measure of long-term outcomes.”

Together, the team will work with the 14 research institutions across the United States to collect data and then examine rates of complications or associated illnesses, as well as how adult patients have accessed health care throughout their lives. The findings may help predict which patients are at greater risk of falling out of routine health care, and when these gaps in care are likely to occur across a patient’s lifespan. The study will also correlate findings with how patients are actually feeling in their everyday lives.

The 14 participating institutions are:

• Ochsner Health
• Children’s Hospital of Philadelphia
• Icahn School of Medicine at Mount Sinai
• University of California – San Francisco
• Nationwide Children’s Hospital
• Duke University Health System
• NYU Grossman School of Medicine
• Nicklaus Children’s Hospital
• Children’s Hospital Colorado
• University of Miami
• Columbia University Irving Medical Center
• Cincinnati Children’s Hospital Medical Center
• Weill Cornell Medical College
• University of Florida

“This unprecedented look at the health of adults living with congenital heart disease allows us to get a full spectrum view by combining clinical data with patient-reported health data,” says Dr. Carton.

The first patient-powered registry for adults with CHD — the Congenital Heart Initiative (CHI) is a key component of this research. Launched with seed funding from Children’s National and the Heart Research Alliance at UCSF, the CHI is led by Dr. John and her team at Children’s National in addition to a broad multi-stakeholder advisory board, including patients. The CHI was co-developed with input from patients, clinicians and researchers and continues to involve these voices in the advancement of the registry. Patients who are recruited for this research will participate via enrollment in the registry, which will allow researchers to ask patients directly about health, wellness and any specific barriers to care. Learn more about CHI’s progress in their first annual report.

“Patients, like myself, are charting a new course and we desire answers to significant questions, as do our providers, about impact of lifelong specialized care, along with improved understanding of the quality-of-life patients experience,” says Leezer. “This project represents a huge step forward towards obtaining answers for the adult CHD community.”

The study also draws on the vast health data resources of PCORnet®, the National Patient-Centered Clinical Research Institute, to conduct this study more efficiently. With health records for 66 million patients available for observational studies, the PCORI-funded PCORnet provides vast scale to power research on conditions affecting even small numbers of people.

“We are confident that this research will yield critical learnings that will empower the community, align resources and spur future innovation to better meet the specialized care needs of this emerging population,” says Mark Roeder, president and chief executive officer for the Adult Congenital Heart Association.

PCORI is an independent, nonprofit organization authorized by Congress in 2010 to fund research that will provide patients, their caregivers and clinicians and other health care decision makers with the evidence-based information needed to make better-informed health care choices.