Stability and Variability of Symptom Burden in Primary Biliary Cholangitis: Insights From a Decade of Follow‐Up

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Abstract
Background and Aims: Primary biliary cholangitis (PBC) frequently impairs health‐related quality of life (HRQOL); however, longitudinal changes in symptoms and their relationship with prognosis remain unclear. We aimed to elucidate the current status of symptoms and HRQOL in Japanese patients with PBC, evaluate 10‐year changes in PBC‐40 scores, and examine associations with long‐term outcomes. Methods: This multicenter observational study comprised three analyses: (i) a 2025 cross‐sectional study assessing associations between clinical parameters, PBC‐40 (symptom indicators), and EQ‐5D‐5 L (HRQOL indicators); (ii) a longitudinal PBC‐40 study comparing scores from 2015 to 2025; and (iii) a longitudinal outcome study evaluating 2015 PBC‐40 scores and clinical parameters in relation to survival through 2025. Results: In 2025%, 20%–50% of all patients ( n = 239) had moderate/severe PBC‐40 scores in each domain. EQ‐5D‐5L scores were significantly lower in women < 59 years versus general population, with multivariable analysis identifying itch, fatigue, and social domains as independent contributors to impaired HRQOL. Across a 10‐year follow‐up, overall significant deterioration was observed in symptoms ( p = 0.0015), itch ( p = 0.0039), and cognitive ( p = 0.0011) domains, while interindividual variability was greatest in itch and emotional and smallest in symptoms. In longitudinal outcome study ( n = 410), no PBC‐40 domain predicted death or liver transplantation. Conclusions: HRQOL in patients with PBC was significantly impaired in younger female. Over 10 years, overall and interindividual changes in domain scores greatly varied, suggesting that symptom burdens are driven by heterogeneous underlying mechanisms. HRQOL impairment represents distinct dimensions of disease impact in PBC, independently of long‐term outcomes.
Funding Information
  • Ministry of Health, Labour and Welfare (JPMH23FC1026)
  • Kowa Company
  • EA Pharma Co., Ltd.

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