Gilead Sciences

This information is intended for US healthcare professionals.

Gilead Sciences

This information is intended for US healthcare professionals.

  2. Primary Biliary Cholangitis
  3. PBC Unmet Needs

Unmet Needs in Primary Biliary
Cholangitis (PBC)

In PBC, quality of life (QoL) and treatment outcomes remain unmet needs, with many patients continuing to experience debilitating symptoms, disease progression, and incomplete biochemical response.1-3

Improving QoL

(Extrahepatic manifestations and symptom response)

Up to ~80% of patients independently experience both pruritus and fatigue, reducing QoL1,2

  • Current guideline-recommended treatments have been shown to inadequately manage PBC symptoms like pruritus, and are often associated with DDIs and significant side effects.4
  • New management strategies are needed to improve outcomes1,2

Extrahepatic manifestations are seen in up to 95% of patients, further burdening patient QoL3,5,6

  • Sjögren's syndrome, thyroid disease, osteoporosis/osteopenia, hypercholesterolemia, and vitamin malabsorption can occur3,5,6

Improving disease outcomes

(Disease progression and biochemical responses)

40%-68% of patients progress to cirrhosis within 4 years without adequate treatment7

Current 1L (1st line) therapy has been shown to be ineffective in reducing ALP in a subset of patients with PBC8

  • Patients without normalized ALP are at an increased risk of disease progression, liver transplant, and death9

The Patient Experience in PBC

The PBC treatment pathway is centered around delivering personalized care or therapeutic decisions tailored to each patient, supporting them at every stage of their lifelong journey. Patients experience a range of emotions, with opportunities to focus on raising urgency for symptom relief and providing PBC management support.10

Personalizing PBC Treatment10

Recently published in Hepatology.

Diagram that illustrates personalizing PBC treatment

Key Challenges in Treating PBC:

HCP (primarily PCP) lack of PBC awareness may cause misinterpreted lab results, overlooked symptoms, and delayed referrals and diagnosis11,12

There is a steep and continuous learning curve for the average patient to overcome in order to remain engaged and participate meaningfully in shared treatment decision-making11

HCPs may tend to prioritize reducing the risk of disease progression while patients aim to alleviate debilitating symptoms, though this is increasingly being viewed as a treatment goal by HCPs as well11

Patients need peer-to-peer support and holistic care to address burdens and challenges not directly improved by treatments11

Patient activation is affected by various barriers, including: Identifying patients that may benefit from changes in disease management, stigma related to perceived alcoholism, access to care, and other social determinants of health11

2L, second-line; ALP, alkaline phosphatase; DDI, drug-drug interaction; ELF, enhanced liver fibrosis; LSM, liver stiffness measurement; PBC, primary biliary cholangitis; TB, total bilirubin; ULN, upper limit of normal.

References:

  1. Onofrio FQ, Hirschfield GM, Gulamhusein AF. A practical review of primary biliary cholangitis for the gastroenterologist. Gastroenterol Hepatol (NY). 2019;15(3):145-154.
  2. Jones D, Carbone M, Invernizzi P, et al. Impact of setanaxib on quality of life outcomes in primary biliary cholangitis in a phase 2 randomized controlled trial. Hepatol Commun. 2023;7(3):e0057. doi:10.1097/HC9.0000000000000057
  3. Wah-Suarez MI, Danford CJ, Patwardhan VR, Jiang ZG, Bonder A. Hyperlipidaemia in primary biliary cholangitis: treatment, safety and efficacy. Frontline Gastroenterol. 2019;10(4):401-408. doi:10.1136/flgastro-2018-101124
  4. European Association for the Study of the Liver. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. J Hepatol. 2017;67(1):145-172. doi:10.1016/j.jhep.2017.03.022
  5. Younossi ZM, Bernstein D, Shiffman ML, et al. Diagnosis and management of primary biliary cholangitis. Am J Gastroenterol. 2019;114(1):48-63. doi:10.1038/s41395-018-0390-3
  6. Chalifoux SL, Konyn PG, Choi G, Saab S. Extrahepatic manifestations of primary biliary cholangitis. Gut Liver. 2017;11(6):771-780. doi:10.5009/gnl16365
  7. Laschtowitz A, de Veer RC, Van der Meer AJ, Schramm C. Diagnosis and treatment of primary biliary cholangitis. United European Gastroenterol J. 2020;8(6):667-674. doi:10.1177/2050640620919585
  8. Lindor KD, Bowlus CL, Boyer J, Levy C, Mayo M. Primary Biliary Cholangitis: 2018 Practice Guidance from the American Association for the Study of Liver Diseases. Hepatology. 2019;69(1):394-419. doi:10.1002/hep.30145
  9. Lammers WJ, van Buuren HR, Hirschfield GM, et al. Global PBC Study Group. Levels of alkaline phosphatase and bilirubin are surrogate end points of outcomes of patients with primary biliary cirrhosis: an international follow-up study. Gastroenterology. 2014;147(6):1338-49.e5; quiz e15. doi:10.1053/j.gastro.2014.08.029
  10. Levy C, Bowlus CL. Primary biliary cholangitis: Personalizing second-line therapies. Hepatology. 2025;82(4):895-910. doi:10.1097/HEP.0000000000001166
  11. Grønkjær LL, Lauridsen MM. Quality of life and unmet needs in patients with chronic liver disease: A mixed-method systematic review. JHEP Rep. 2021;3(6):100370. doi:10.1016/j.jhepr.2021.100370
  12. Shamaa O, Ahmed A, Rupp L, Trudeau S, Gordon SC. Beyond the Surface: Unveiling Hidden Hurdles to Primary Biliary Cholangitis Care. Cureus. 2024;16(7):e64753. doi:10.7759/cureus.64753