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

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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