Primary Biliary Cholangitis

Primary biliary cholangitis (PBC), also known as primary biliary cirrhosis, is a rare and chronic autoimmune disease that causes the small bile ducts in the liver to become inflamed, damaged and ultimately destroyed.1

For those with PBC, bile builds up in the liver over time and slowly damages cells. This ultimately results in scarring of the liver, or cirrhosis. As cirrhosis progresses, scar tissue builds up, and the liver stops functioning. While PBC progresses slowly, it may lead to life-threatening complications, especially after cirrhosis develops.1

The symptoms of PBC are often slow to develop, and in some people, may be nonexistent. This absence of symptoms is especially common in the early stages of the disease. The most common symptom of PBC is pruritus, or itching on the arms, legs and back. Fatigue, jaundice, fluid build-up in the ankles and abdomen, darkening of the skin, and collection of fatty deposits in the skin around the eyes can also occur. Jaundice, a yellowish color to the skin or white part of the eyes, is a gauge for measuring disease progression.1

While the cause of PBC is unknown, some believe it may be related to a weakened immune system. PBC is not a genetic disease, but is more common among siblings and in families where one member has been diagnosed. The disease is ultimately diagnosed by a series of tests, but since many patients don't have symptoms, PBC is often identified unintentionally (through routine liver blood tests).1

Maintaining a healthy lifestyle that includes eating a reduced sodium diet, drinking ample amounts of water, and exercising may help relieve or prevent some PBC symptoms. PBC is most often treated with ursodeoxycholic acid, a bile acid that is produced naturally by the body, which is also known as ursodiol or UDCA.1

Currently, ursodeoxycholic acid is prescribed only in solid forms, such as a tablets or capsules.2 We are exploring the potential of a liquid formulation of ursodeoxycholic acid that would provide healthcare professionals with an alternative for PBC patients who have difficulty swallowing. Learn more about this formulation.

The following organizations may be a resource for information and support for people with primary biliary cholangitis and those around them:

Global Genes

Global Genes is a non-profit patient advocacy organization working to eliminate the challenges of rare disease by building awareness, educating the global community, and providing critical connections and resources that equip advocates to become activists for their disease.  Global Genes promotes the needs of the rare disease community under a unifying symbol of hope – the Blue Denim Genes Ribbon™. Numerous tools, resources and educational events can be found on the Global Genes website www.globalgenes.org.

National Organization for Rare Disorders

The National Organization for Rare Disorders (NORD) is a unique federation of voluntary health organizations dedicated to helping people with rare "orphan" diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment and cure of rare disorders through programs of education, advocacy, research and service. In the US, NORD is also the official sponsor of Rare Disease Day, an annual global observance that raises awareness of rare diseases and elevates them into the public consciousness as an important health issue that cannot be ignored.

EURORDIS

EURORDIS is a non-governmental patient-driven alliance of more than 700 rare disease patient organisations in more than 60 countries. EURORDIS strives to build a strong pan-European community of patient organisations and people living with a rare disease, to be their voice at the European level, and - directly or indirectly - to fight against the impact of rare diseases on their lives. EURORDIS leads global efforts for Rare Disease Day and the online social platform for people living with or affected by a rare diseases RareConnect. EURORDIS represents the voice of an estimated 30 million people living with a rare disease in Europe. Follow @eurordis or see the EURORDIS Facebook page. For more information visit: www.eurordis.org.

 

 

  1. American Liver Foundation. Primary Biliary Cholangitis (PBC, Primary Biliary Cirrhosis). Available at: http://www.liverfoundation.org/abouttheliver/info/pbc/. Last accessed July 28, 2016.
  2. Setchell, et al. Aliment Pharmacol Ther 2005; 21: 709–721.