This FAQ delves into the promising trends and developments in Biliary Cholangitis (PBC) research. DOWNLOAD THE FULL REPORT HERE

What is Primary Biliary Cholangitis (PBC)?

PBC is a chronic inflammatory hepatobiliary illness characterized by T-cell lymphocyte-mediated destruction of the interlobular bile ducts. If left untreated, it can lead to biliary cirrhosis and liver failure.

Who is most commonly affected by PBC?

PBC predominantly affects women, with more than 90% of patients being female. It is typically diagnosed between the ages of 30 and 40, and the female-to-male ratio is approximately 4-6:1.

What are the early signs and symptoms of PBC in women?

Early signs and symptoms in women may include tiredness, pruritus (itching), stomach pain, jaundice, abnormal liver function tests, and elevated alkaline phosphatase levels.

How does PBC manifest differently in men?

PBC in men is often diagnosed later in life and is associated with a weaker response to ursodeoxycholic acid (UDCA), quicker progression to cirrhosis, higher liver-related mortality rates, and an increased risk of hepatocellular carcinoma (HCC).

What are the standard care measures for PBC patients?

Standard care for PBC patients includes hepatitis A and B vaccination for those without serologic protection. Additionally, pneumococcal vaccination and general population immunizations (e.g., influenza) are recommended for individuals with chronic liver disease. Patients are also advised to limit alcohol consumption.

What are the approved treatments for PBC?

Ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) are the two FDA-approved medications for PBC. UDCA is the first-line therapy, while OCA is the second-line option. Research is ongoing to explore other potential disease-modifying therapies.

How many ongoing clinical trials for PBC treatments?

There have been approximately 140 ongoing clinical trials for PBC since 2018, with a significant number of trials conducted in the Asia-Pacific region, the United States, and Europe.

What regions are prominent in PBC clinical trials?

The Asia-Pacific region, particularly Mainland China, has a substantial presence in PBC clinical trials. The United States, the United Kingdom, and France are prominent in Europe. The Rest of the World (ROW) also contributes moderately to clinical trial activity.

How does trial recruitment differ among regions?

Single-country trials in the Asia-Pacific region have shorter median recruitment durations compared to Europe and the US. The APAC region stands out with remarkably accelerated recruitment rates, making it a hub for efficient clinical trial enrollment.

How has investment in PBC research and development varied across regions?

The United States and China have been leading in venture funding for PBC research. Investments have shown dynamic patterns, with fluctuations in funding levels from year to year. In 2023, the United States and China saw significant investments in PBC research.

Are there superior medications for PBC treatment?

There is currently no evidence that one medication is superior to another in PBC treatment. More longitudinal clinical studies and research into the disease's pathophysiology are needed to identify new treatment targets.

What are the potential future therapies for PBC?

Several potential future therapies for PBC are being explored, including peroxisome proliferator-activated receptor and FXR agonists are being considered for disease-modifying therapies due to their potential to improve liver fibrosis. Additionally, ongoing investigations are exploring the potential of fibroblast growth factor-19 agonists and NOX inhibitors. Research in these areas may lead to additional treatment options in the future.