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Navigating Life With PBC: Empowering Those Affected by a Rare Autoimmune Disease

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In approximately 1 in 1000 women worldwide over the age of 40 and in the US, as many as 65 out of every 100,000 women are diagnosed with a rare, autoimmune disease that can cause significant liver damage and potentially lead to liver failure if left untreated.1,2 This disease is known as primary biliary cholangitis (PBC).3 Although PBC is more common in women, we now know that more men than previously thought also have this disease.4

Many people may not experience symptoms in the early stages of the disease.2 Unfortunately, there is also no cure.2,5 If this chronic liver disease is left untreated, it can progress to cirrhosis.2

The Path to PBC Diagnosis and Treatment

The journey to a PBC diagnosis can be long and challenging, especially when there may not be any signs of the disease that others can see.6 For many patients, the path to a PBC diagnosis can involve numerous testing and procedures.7

First-line and second-line treatment options are available to patients. After 1 year on first-line therapy, one’s health care provider will assess treatment goals by looking at biochemical response.3,8 Some patients don’t meet treatment goals—this is known as an inadequate response to therapy. In such cases, several second-line treatments are available for appropriate patients who qualify.3,8

Routine liver blood tests can reveal abnormalities.2 If PBC is suspected by one’s doctor, an antimitochondrial antibody (AMA) test is generally used to confirm a PBC diagnosis.2 For people living with PBC, some treatment goals include normalizing or improving a protein called alkaline phosphatase (ALP) and managing symptoms of the disease, such as pruritus.9-10 A treatment plan that helps sustain ALP levels and more effectively manages symptoms may help improve disease outcomes.9-10

Living With PBC

Symptoms can range from mild to severe and can develop slowly over 5 to 20 years.11 As the disease progresses, there is an increased risk of inflammation of the bile duct and scarring of the liver that may cause serious complications.11

More than half of people diagnosed with PBC are asymptomatic and not all symptoms of PBC are visible.12 The symptoms that commonly appear—such as pruritus (chronic itch) and fatigue—are often invisible to others and may negatively affect daily life.9 The debilitating itchy skin that may be associated with PBC can have a profoundly negative impact on a person’s daily life and those around them and may also be associated with sleep disturbance, fatigue, and depression.9

For patients living with PBC, it is important to connect with others who have similar experiences who can provide crucial support, in addition to support from loved ones.13-14 It is also important to have a supportive care team that listens attentively, thoroughly explains the treatment process, and presents all available options.13-14

Looking Ahead

As research and awareness of PBC continue to grow, there is hope for those living with the disease.8,15 Advancements in medical science aim to improve both the understanding and management of PBC, offering patients new avenues for care and support.8,15

After receiving a PBC diagnosis, it’s essential for patients to maintain close communication with their care team. Regular check-ins with healthcare providers ensure that any changes in symptoms are promptly addressed and that the treatment plan is continually optimized.16 Beyond medical management, discussing ways to improve the patient’s daily life is key. Consider lifestyle adjustments for the patient, such as adopting a healthy diet and engaging in regular physical activity, along with treatment, which can help mitigate the progression of PBC.16

By having the patient be proactive and informed, they can work alongside their care team to manage PBC effectively.2

This content is sponsored by Gilead Sciences.

References:

  1. 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
  2. American Liver Foundation. Primary biliary cholangitis. Accessed September 30, 2024. Available at: https://liverfoundation.org/liver-diseases/autoimmune-liver-diseases/primary-biliary-cholangitis-pbc/
  3. Levy C, Bowlus CL, Carey E, et al. A real-world observational cohort of patients with primary biliary cholangitis: TARGET-primary biliary cholangitis study design and rationale. Hepatol Commun. 2018;2(5):484-491. doi:10.1002/hep4.1173
  4. Shaker M, Mansour N, John BV. Primary biliary cholangitis in males: pathogenesis, clinical presentation, and prognosis. Clin Liver Dis. 2022;26(4):643-655. doi: 10.1016/j.cld.2022.06.008
  5. Bernal RB, Ferrigno B, Morales EM, et al. Management of primary biliary cholangitis: current treatment and future perspectives. Turk J Gastroenterol. 2023;34(2):89-100. doi:10.5152/tjg.2023.22239
  6. 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
  7. Mayo Clinic. Primary biliary cholangitis diagnosis. Accessed September 17, 2024. Available at: https://www.mayoclinic.org/diseases-conditions/primary-biliary-cholangitis/diagnosis-treatment/drc-20376880
  8. Bonder A. Current and emerging treatment options for primary biliary cholangitis. Gastroenterol Hepatol (N Y). 2020;16(7):363-365
  9. Sivakumar T, Kowdley KV. Anxiety and depression in patients with primary biliary cholangitis: current insights and impact on quality of life. Hepat Med. 2021;13:83-92. doi: 10.2147/HMER.S256692
  10. 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. 2018;0(0):1-26. doi:10.1002/hep.30145
  11. Mayo Clinic. Primary biliary cholangitis. Accessed August 29, 2024. Available at: https://www.mayoclinic.org/diseases-conditions/primary-biliary-cholangitis/symptoms-causes/syc-20376874
  12. Prince MI, Chetweynd A, Craig WL, et al. Asymptomatic primary biliary cirrhosis: clinical features, prognosis, and symptom progression in a large population based cohort. Gut. 2004;53(6):865-870. doi:10.1136/gut.2003.023937
  13. National Organization for Rare Disorders. Primary biliary cholangitis organization. Accessed September 30, 2024. Available at: https://rarediseases.org/organizations/primary-biliary-cholangitis-organization/
  14. Global Liver Institute. Primary biliary cholangitis (PBC) externally-led patient-focused drug development (EL-PFDD). Accessed September 30, 2024. Available at: https://globalliver.org/wp-content/uploads/2023/02/PBC-EL-PFDD.pdf
  15. Bahar R, Wong KA, Liu CH, Bowlus CL. Update on new drugs and those in development for the treatment of primary biliary cholangitis. Gastroenterol Hepatol (N Y). 2018;14(3):154-163
  16. Cleveland Clinic. Primary biliary cholangitis (PBC): living with this chronic disease. Accessed September 30, 2024. Available at: https://health.clevelandclinic.org/living-with-pbc

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