PCORI Awards $13 Million for Study of Symptom Care in Two Rare Liver Diseases

The non-profit Patient-Centered Outcomes Research Institute awarded a five-year, $13 million grant to The University of North Carolina at Chapel Hill to test new ways of helping adults manage the often-overlooked symptoms of two rare chronic liver diseases.

The award remains pending PCORI’s completion of business and programmatic review and the issuance of a formal contract.

The study, known as TRANSFORM-EMPOWER, will compare a self-directed digital mind-body program with the same program paired with clinician-led virtual group psychotherapy. Researchers will focus on outcomes that can affect daily life, including mental health, fatigue and other symptoms in people living with primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC).

PBC and PSC are cholestatic liver diseases, meaning they interfere with the normal flow of bile. Both can bring a long and unpredictable disease course, while symptoms such as severe fatigue, itching, abdominal discomfort and emotional distress can disrupt work, relationships and quality of life. PSC can also cause jaundice, diarrhea and episodes of fever; some people may have no symptoms at diagnosis.

Despite those burdens, access to behavioral-health professionals who understand rare liver disease can be limited, particularly for patients outside major medical centers.

“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 Donna Evon, the study’s principal investigator and a professor of medicine in UNC School of Medicine’s Division of Gastroenterology and Hepatology.

Evon said the project will establish hepatologist-psychologist teams at every study site to examine “blended therapy,” combining liver-specialty care with psychological support.

The study plans to enroll and randomly assign 600 adults with PBC or PSC at 17 liver centers in the United States and Canada. One group will use a digital mind-body symptom-management program on its own; the other will receive the program plus clinician-guided virtual group psychotherapy. The research will assess not only which approach better improves symptoms that matter to patients, but also whether the interventions can realistically be incorporated into routine care across different liver centers.