PCORI Awards $13M to UNC to Study Innovative Symptom Management Interventions for Rare Cholestatic Liver Diseases

UNC School of Medicine's Donna Evon, PhD, will lead a multi-center study evaluating a mind-body digital health program with and without clinician-guided psychotherapy to improve mental health, fatigue and other symptoms for adults with rare cholestatic liver diseases.

The Patient-Centered Outcomes Research Institute (PCORI) has announced a $13 million funding award to the University of North Carolina at Chapel Hill over five years to study innovative symptom management interventions for patients with rare cholestatic liver diseases.

The study is led by principal investigator Donna Evon, PhD, professor of medicine in the Division of Gastroenterology and Hepatology at the UNC School of Medicine, and co-principal investigator Puneeta Tandon, MD, professor of medicine at the University of Alberta. The researchers will compare a mind-body symptom management digital health program used alone and the same program combined with clinician-guided virtual group therapy. They will examine how these approaches affect mental health, fatigue, and other symptoms in people with cholestatic liver disease.

The project, called TRANSFORM-EMPOWER, incorporates a comparative clinical effectiveness trial concurrently with observing integration in real world clinical practice. It will randomize 600 adults with primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) from 17 liver centers across the US and Canada. The project was developed in close collaboration with PSC and PBC patient advocacy organizations. UNC faculty involved in the project include Xianming Tan, PhD, Micha Belden, Neil Shah, MD, Michael Kappelman, MD, Ethan Basch, MD, Darren DeWalt, MD, Jacquie Halladay, MD, Kevin Angstrom, PhD, and Saif Khairat, PhD.

"Many patients with PBC and PSC live with burdensome symptoms and uncertainty about the future, which can take a real toll on their quality of life," said Evon. "They lack access to mental health providers who understand the challenges of living with these diseases. I am excited to have created new hepatologist-psychologist teams at each site to evaluate the added benefits of 'blended therapy', a model that is currently underutilized at academic medical centers."

The research team will study whether adding clinician-guided virtual group psychotherapy to an accessible digital health program provides more benefit than using a self-directed digital program alone. The findings could improve symptom management not only for people with rare liver diseases, but also for patients with other rare or chronic conditions who face similar barriers to behavioral health care.

"This study exemplifies the kind of rigorous, patient-centered research that is needed to improve care for people living with chronic and rare diseases," said Darren A. DeWalt, MD, Distinguished Professor and Chair of the Department of Medicine at the UNC School of Medicine and a co-investigator on the study. "By combining a large comparative effectiveness trial with a robust evaluation of implementation across 17 liver centers in the United States and Canada, we will learn not only whether these interventions improve outcomes that matter to patients, but also how they can be successfully integrated and sustained in clinical practice."

The funding award to The University of North Carolina at Chapel Hill has been approved pending completion of PCORI's business and programmatic review and issuance of a formal award contract.

"Under the outstanding leadership of Dr. Donna Evon, this study will generate vital evidence on scalable models of care that can improve mental health, fatigue, and quality of life for patients with PSC and PBC, while informing healthcare delivery for many other chronic diseases. We are proud that UNC is leading this important international collaboration focused on outcomes that matter most to patients and their families," said Millie Long, MD, MPH, vice chief of education and director of the fellowship program in the Division of Gastroenterology and Hepatology at the UNC School of Medicine.

PCORI is a nonprofit organization with a mission to fund patient-centered CER designed to provide patients and those who care for them with evidence to make better-informed healthcare decisions.