Pruritus Increases HRQoL Burden in PBC, Underscoring Need for Effective Treatments

People with primary biliary cholangitis (PBC) and moderate-to-severe pruritus experience a greater health-related quality of life (HRQoL) burden and reduced activity compared to those with mild-to-no pruritus, according to findings from a recent study.1

Patients with PBC were recruited from the PBCers Organization, the largest PBC online support group in the US, and through physician panels. Results showed those with pruritus experienced a significantly greater negative impact on HRQoL across a range of domains, including those related to itch, fatigue, and social and cognitive function. Of note, those with pruritus reported infrequent use of treatments targeting itch, with minimal or no relief.1

In 2024, the US Food and Drug Administration approved 2 new second-line treatment options for PBC: Ipsen’s elafibranor (Iqirvo) and Gilead’s seladelpar (Livdelzi). However, even with these new therapies available in combination with ursodeoxycholic acid (UDCA) in adults who have had an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA, many patients continue to experience debilitating itch.2,3

“Given the limited research in this area in the US, obtaining patients' perspectives regarding the burden and unmet treatment needs of PBC-related cholestatic pruritus is critical to raising awareness of their experiences, HRQoL, and psychosocial needs,” Cynthia Levy, MD, a professor of clinical medicine and hepatology at the University of Miami Miller School of Medicine, and colleagues wrote.1 “Capturing the patient's voice may further illuminate the challenges they face, providing valuable insights that could guide more effective treatment and management strategies.”

To assess the impact of pruritus on HRQoL and treatment experiences of people with PBC, investigators recruited US participants from the PBCers Organization and through physician panels. Patients with a diagnosis of PBC were eligible to participate if they were ≥ 18 years of age, living in the US, self-reported that they were currently taking an FDA-approved treatment for PBC (at the time of the study, either UDCA or obeticholic acid), and provided study informed consent.1

Based on the Pruritus NRS completed during screening, participants were categorized into 2 groups. Patients with a Pruritus NRS score of 0–3 were allocated to the No/Mild Pruritus (NMP) group, and patients with a Pruritus NRS score of 4–10 were allocated to the Moderate/Severe Pruritus (MSP) group.1

Participants additionally completed the 5-D Itch, PBC-40, EQ-5D-5L, Chronic Liver Disease Questionnaire (CLDQ-PBC), Work Productivity and Activity Impairment (WPAI), and Functional Assessment of Chronic Illness Therapy – Fatigue Scale, Version 4 (FACIT-Fatigue) questionnaires, and PBC treatment experiences questions. A subset of participants from the MSP group was also asked to respond to qualitative voice questions by calling into an automated voice response system.1

A total of 159 individuals were recruited and completed the eligibility screener, of whom 122 (77%) met study eligibility criteria and 90 (57%) completed the study, including 40 in the NMP group and 50 in the MSP group.1

Compared with the NMP group, investigators noted the MSP group had significantly worse 5-D Itch total scores (P <.001) and EQ-5D Index scores (P = .005). Additionally, the MSP group had significantly worse PBC-40 mean scores in the Symptom, Itch, Fatigue, Cognitive, and Social domains (all P <.05); but not in the Emotional domain (P = .239).1

The MSP group also had worse total FACIT-Fatigue and CLDQ-PBC scores (both P <.001) compared to the NMP group. Investigators pointed out the MSP group also had significantly lower CLDQ-PBC scores across the Diet, Emotional, Fatigue, Itch, Sleep, and Symptoms domains (all P <.05).1

Further analysis revealed the MSP group reported significantly greater activity impairment due to PBC (57% vs 36%; P <.001). The MSP group also had a lower employment rate (42%) than the NMP group (53%).1

While most patients in both groups were treated with ursodeoxycholic acid and/or obeticholic acid, fewer than 25% of the MSP group was receiving treatment for pruritus.1

“This study expands on prior analyses and provides further evidence that people with PBC, representing diverse racial and ethnic backgrounds, who have moderate-to-severe pruritus experience reduced activity and a greater HRQoL burden compared to those with mild/no pruritus,” investigators concluded.1 “Despite this, individuals with PBC who experience this symptom report infrequent use of treatments targeting pruritus (itching), with minimal or no relief from their itch. There is a clear unmet need for safe and effective treatments that address pruritus and improve associated HRQoL outcomes.”

References

1. Levy C, Younossi ZM, Mayo MJ, et al. Assessment of PBC Pruritus From the ITCH-E Study: Quality of Life, Productivity, and Treatment Experiences. Liver International. https://doi.org/10.1111/liv.70276

2. Brooks A. FDA Grants Accelerated Approval to Elafibranor (Iqirvo) for PBC. HCPLive. June 10, 2024. Accessed August 26, 2025. https://www.hcplive.com/view/fda-grants-accelerated-approval-to-elafibranor-iqirvo-for-pbc

3. Brooks A. FDA Grants Accelerated Approval to Seladelpar (Livdelzi) for Primary Biliary Cholangitis. HCPLive. August 14, 2024. Accessed August 26, 2025. https://www.hcplive.com/view/fda-grants-accelerated-approval-to-seladelpar-livdelzi-for-primary-biliary-cholangitis