SAN DIEGO—Children with autoimmune liver disease and their caregivers face numerous challenges, but their health-related quality of life has not been well studied. A new prospective, longitudinal study from the Cincinnati Children’s Hospital Medical Center and the Autoimmune Liver Disease Network for Kids shines a light on the issue, identifying trends in medication use and disease complications.

Among the findings, autoimmune overlap syndrome was associated with the highest quality-of-life scores, and having complications was associated with better school functioning.

“Our study found that participants with overlap syndrome reported higher quality-of-life scores than participants with [autoimmune hepatitis] or [primary sclerosing cholangitis], and participants with at least one disease complication reported better school functioning,” said investigator Rebecca Farr, MD, a clinical fellow in gastroenterology, hepatology and nutrition at Cincinnati Children’s Hospital Medical Center, who presented the study at The Liver Meeting 2024. “We postulate that these patients may be receiving greater support secondary to their more complex disease states.”

The researchers conducted a multicenter survey of 162 pediatric and young adult patients that assessed four domains: physical, emotional, social and school. The sample was 54% female and 86% white, with a median age at diagnosis of 13 years. The disease type was autoimmune hepatitis (AIH) in 62% of cases, primary sclerosing cholangitis (PSC) in 19%, and overlap syndrome in 19% of patients. Inflammatory bowel disease occurred in 31% of participants.

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Participants filled out the Pediatric Quality of Life Inventory three times at least six months apart. There were several interesting findings, according to Dr. Farr. Although the patient surveys showed no association between disease type and quality of life, there were statistically significant differences in AIH, PSC and overlap categories reported by caregivers with respect to each of the four domains and total score (P=0.02). The highest scores were in the overlap group, whereas scores for the AIH and PSC groups were lower and generally similar.

“Interestingly, we found that participants taking azathioprine reported lower social functioning, while those taking prednisone and their caregivers reported improved emotional functioning,” Dr. Farr said. “We postulate the side effects of azathioprine may be playing a role in decreasing quality of life, while prednisone may be helping to decrease disease activity, thereby improving quality of life.”

Patients taking azathioprine had a mean score of 87 in the social domain, whereas those who did not had a mean score of 92 (P=0.01). In contrast, Dr. Farr explained, patients taking prednisone reported significantly higher scores in the emotional domain (83) than those who did not (78) (P=0.04), as did caregivers (80 vs. 73; P=0.01). She said the finding was “very surprising, as we all typically think of steroids as negatively affecting mood. The caregivers and participants taking prednisone also reported higher psychosocial scores” (83 vs. 79; P=0.07 in participants and 81 vs. 76; P=0.03 in caregivers).

The researchers also examined fatigue and found that “participants with fatigue reported statistically lower scores in the physical and school domains, while their caregivers reported statistically lower scores in all domains.” Pruritus resulted in worse patient-reported quality of life in the physical, emotional and psychosocial domains, but among caregivers, it led to a significant difference only in the total score.

“It is quite interesting that caregivers report more influence of fatigue on quality of life than the participants, while in the case of pruritus, it is the opposite,” Dr. Farr said.

The results are a welcome contribution to the literature, said Jim Squires, MD, who co-moderated the session with Evelyn Hsu, MD. “The concept of ‘ignorance is bliss’ doesn’t apply to these patients. Even though historically we haven’t asked [about quality of life], it doesn’t mean that we shouldn’t,” said Dr. Squires, an associate professor of pediatrics at the University of Pittsburgh School of Medicine. “I think it underscores the shortage of mental health care providers that I think we critically need to pair with any shortcomings that we see in the quality of life of our families and patients with these chronic illnesses. We’re going to need to eventually come to a space where we can then refer them to resources to begin to address some of the issues that we’re uncovering.”

The findings also highlight the need to focus on more than just the health of the liver but the patient in general, said Dr. Hsu, a professor of medicine at the University of Washington School of Medicine and the division head of gastroenterology and hepatology at Seattle Children’s Hospital. “We have to ask these questions in these really critical times of development: How are they coping? Because that predicts how they will cope for this lifetime of disease that they have,” she said. “It used to be that we’re trying to cure the disease and increase their survival, but now it’s really about increasing the quality of their survival, so that they go on to be as productive members of society as possible in the coming decades.”

—Jim Kling

Drs. Farr, Hsu and Squires reported no relevant financial disclosures.

This article is from the May 2025 print issue.