Introduction

Gastro-oesophageal varices (GOV) can occur in early stage primary biliary cholangitis (PBC), making it difficult to identify the appropriate time to begin screening with oesophageo-gastro-duodenoscopy (OGD). This proves the utility of a clinical tool to predict the probability of finding GOV in PBC patients such as the Newcastle Varices in PBC (NVP) Score.

Aims & Methods

Our aim was to assess the performance of this score in predicting GOV in PBC patients. We conducted a retrospective study analyzing clinical data of 72 PBC patients treated with Ursodeoxycholic acid (UDCA). The presence of GOV was noted as well as the occurrence of variceal bleeding. The NVP score was calculated for all the patients who underwent OGD. The performance of the score was assessed using ROC curves.

Results

Seventy-two patients were included with a mean age of 53.5 years. UDCA at the dose of 13-15 mg/kg/day was prescribed for all the patients. The median follow-up time was 9 years. An OGD was done for 69 patients (95.8%). Esophageal varices were found in 34 patients (47.2%). They were grade 1, 2 and 3 in respectively 12.5%, 29.2% et 5.6% of the patients. Gastric varices were present in 5 patients (6.9%). A quarter (25%) of the patients had a variceal bleeding during the follow-up time. The analysis of ROC curves showed that the NVP score had a good performance to predict the development of GOV in PBC patients (AUC=0.914, p<0.001).

Conclusion

According to our study, the NVP score is an inexpensive and non-invasive tool that accurately predicts GOV in PBC. Thus, it can be considered a substitute for OGD for GOV screening in PBC patients. Further studies are warranted to confirm these results.

Introduction

Aims & Methods

Results

Conclusion

Introduction

The response to ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC) varies widely. Identifying patients at risk for non-response early in the disease course is crucial for anticipating the need for second-line therapies.

Aims & Methods

This monocentric retrospective study included 83 patients with PBC who were treated with ursodeoxycholic acid (13–15 mg/kg/day). Therapeutic response was assessed after one year using the Paris II criteria. The primary objective was to identify factors associated with non-response to UDCA.

Results

The mean age at diagnosis was 55.6 years (range: 27–82), with a clear female predominance. The most common clinical presentations were fatigue (34.9%) and pruritus (22.9%). Additionally, 25 patients (30.1%) had cirrhosis at diagnosis. Autoimmune diseases were observed in 60.2% of cases, primarily autoimmune thyroiditis. At one year, 30.1% of patients were classified as non-responders to UDCA treatment. Non-responders were older at diagnosis (p = 0.026), more frequently had cirrhosis (p < 0.01), and exhibited a more severe biological profile at diagnosis, including elevated total bilirubin (p = 0.013), hypoalbuminemia (p < 0.01), thrombocytopenia (p = 0.016), and a decreased prothrombin time (p = 0.022). However, initial clinical symptoms (fatigue, pruritus, arthralgia) and autoimmune profiles (specific antibodies, associated autoimmune diseases) were not associated with treatment response. A statistically significant positive correlation was found between non-response to UDCA and continuous prognostic scores (GLOBE, UK-PBC, Mayo Risk; p < 0.01), as well as non-invasive fibrosis markers (FIB-4: p = 0.032; FibroIndex: p = 0.015; GPR: p = 0.015).

Conclusion

In PBC, the factors driving non-response to UDCA do not appear to be related to clinical symptoms or autoimmune profiles, but rather to an early biological signature. A combined approach, incorporating prognostic scores and non-invasive fibrosis markers, could help identify at-risk patients sooner and guide treatment decisions.

Disclosure

None.

Introduction

The current universal standard of care in primary biliary cholangitis (PBC) is ursodeoxycholic acid (UDCA), although its clinical benefit in PBC has never been reported in absolute measures.

Aims & Methods

This cohort aimed to assess the prevalence of incomplete response to UDCA and determine non-response predictors.
Patients with PBC as main diagnosis were included from the hospital's centralized electronic medical records system, over a 10-year period. Overlap syndromes (PBC-Auto-Immune Hepatitis) were also included. Main endpoints included incomplete response to UDCA treatment according to Paris I, Paris II criteria, and Globe score.

Results

A total of 50 patients were included in this study. The mean age at diagnosis was 52.74 years (24-73 years) and 92% females. Only one patient was asymptomatic at onset and 82% had positive anti‐mitochondrial antibodies. All patients were treated with ursodeoxycholic acid (UDCA). Corticosteroids and immunosuppressive therapies were associated in 26% of cases. Incomplete response to UDCA was observed in 28% and 34% of patients according to Paris I and II criteria, respectively. Non-response predictors were poor therapeutic adherence, Overlap syndrome and hepatocellular carcinoma in 14%, 6% and 4% respectively. Five patients were lost to follow-up and five cases died from cirrhosis ’complications. The median ΔGLOBE score was 0.42 (IQR: -1.58 to 3.92). The 5, 10 and 15 years liver transplantation-free survival (median) was: +10%, +7.38% and +5% respectively. Liver transplantation (LT) was considered in one patient with excellent outcomes at last follow-up.

Conclusion

A considerable proportion of patients showed incomplete response to UDCA treatment according to Paris II criteria. Early identification of patients at risk of incomplete response could improve patient management and overall disease prognosis.

References

- Treatment response to ursodeoxycholic acid in primary biliary cholangitis: A systematic review and meta-analysis. DOI: 10.1016/j.dld.2022.12.010
- Pretreatment prediction of response to ursodeoxycholic acid in primary biliary cholangitis: development and validation of the UDCA Response Score. doi: 10.1016/S2468-1253(18)30163-8. Epub 2018 Jul 13.

Introduction

Primary biliary cholangitis (PBC) is a chronic autoimmune inflammatory disease of the intrahepatic bile ducts. It is characterized by chronic cholestasis, the presence of specific auto immune antibodies, and distinctive histological lesions. PBC is frequently associated with other autoimmune diseases.

Aims & Methods

This study aims to describe the clinical, biological, morphological, and histological characteristics of PBC patients with associated autoimmune diseases, as well as the therapeutic approaches used.
This was a descriptive retrospective study conducted in the Medicine C of Hepatogastroenterology Department at Ibn Sina Hospital in Rabat, including 62 patients with PBC and associated autoimmune diseases between 2010 and 2025.

Results

Among 130 patients with PBC, 62 had an associated autoimmune disease, representing a prevalence of 47%. The mean age was 47 years, with a sex ratio of 1M/9F. The most frequent symptoms were jaundice in 45.2% (n=28) of cases, asthenia in 41.9% (n=26), and pruritus in 35.5% (n=22). Clinical examination revealed signs of portal hypertension in 48.4% (n=30) of patients, jaundice in 51.6% (n=32), and scratch lesions in 19.4% (n=12).
Abdominal ultrasound showed liver with features of chronic liver disease in 71% (n=44) of patients, splenomegaly in 41.9% (n=26), ascites in 37.1% (n=23), and collateral venous circulation in 32.3% (n=20).
Biological abnormalities included cytolysis in 71% (n=44) of cases, cholestasis in 38.7% (n=24), and hepatocellular insufficiency in 22.6% (n=14). Regarding the autoimmune profile, 62.9% of PBC cases were seropositive, with anti-mitochondrial antibodies type 2 detected in 45% (n=28), anti-Sp100 antibodies in 19% (n=12), and anti-Gp210 antibodies in 16% (n=10) of patients.
Liver biopsy was performed in 40 patients and the Scheuer stages were classified as follows: stage 1 in 12.9% (n=8), stage 2 in 29% (n=18), stage 3 in 19.4% (n=12) and stage 4 in 3.2% (n=2) of cases.
Fibrosis assessment was carried out in 53 patients, using transient elastography (FibroScan) in 13 cases and liver biopsy in 40 cases. The fibrosis stage distribution was as follows: F3 in 38.7% (n=24), F2 in 25.8% (n=16), F4 in 11.2% (n=7), F1 in 8% (n=5), and F0 in 1.6% (n=1) of cases.
The main associated autoimmune diseases were autoimmune hepatitis in 40.3% (n=25) of cases and autoimmune thyroiditis in 40.3% (n=25), followed by Sjögren's syndrome in 21% (n=13), type 1 diabetes in 8.1% (n=5), and celiac disease in 4.8% (n=3). Less frequent associations included juvenile polyarthritis, Graves' disease, and vitiligo (each in 3.2% (n=2) of cases), as well as scleroderma and Addison's disease (each in 1.6% (n=1) of cases). Among these patients, 29% (n=18) had two associated autoimmune diseases, and 6.5% (n=4) had three.
All patients received ursodeoxycholic acid (UDCA) at doses of 13-15 mg/day, along with specific treatments for concomitant autoimmune diseases.
Treatment response assessment using the Paris II score showed a good response in 62% (n=38) of patients and a partial response in 38% (n=24) of patients.

Conclusion

This study confirms the high prevalence of autoimmune diseases associated with PBC, particularly autoimmune hepatitis and thyroiditis, with a strong female predominance. The most frequent stages of fibrosis were F2 and F3, highlighting the moderate to advanced disease progression at diagnosis in many patients. It emphasizes the importance of systematic screening for these comorbidities to ensure optimal management.

Introduction

Pruritus is common in primary biliary cholangitis (PBC) and can significantly impact patients' quality of life (QoL). Cholestyramine is recommended as the first-line treatment option for pruritus in PBC, but tolerability, inconvenient administration, and limited efficacy data restrict its use. Patient-focused drug development can help address this unmet need, but an understanding of patients' perspectives on pruritus and treatments is required. This study conducted semi-structured qualitative patient interviews to explore patients’ experiences, expectations, and concerns with regards to pruritus and treatments.

Aims & Methods

Semi-structured virtual interviews were designed following a literature review and conducted with patients from the USA, Germany, UK, and Italy. Eligible patients self-reported a PBC diagnosis and had a worst itch score of ≥4 on a 0–10 numerical rating scale (NRS) in the past 2 months. Interviews had four parts:
1) pruritus experience,
2) treatment desires and concerns,
3) treatment experience,
4) hypothetical choice questions that required patients to make trade-offs between two options.
Responses were used in an inductive thematic analysis and visually summarized.

Results

Ten patients per country were interviewed (N=40); most were female (78%) and mean (standard deviation) age was 52.5 (15.2) years. For 53% (n=21), pruritus had started within the last 3 years. On the NRS, patients rated their pruritus over the last 2 months on average as mean 5.4 (range 2–10), and worst itch as mean 7.1 (range 4–10). Key themes that emerged from patient discussions were visualized with a concept map. Pruritus was widespread over patients’ bodies, persistent despite scratching, and described as ‘crawling’, ‘burning’ and ‘nettles’. Pruritus impacted social interactions, sleep quality, appearance, emotional stability, and work productivity. Many patients wished for complete resolution, but primarily wanted treatment to reduce pruritus severity (60%; n=23). Of those who had ever received cholestyramine (n=12), 62% reported a repulsive taste and texture; 50% mentioned concerns about interactions between cholestyramine and PBC medication. From five potential pruritus medication side effects, diarrhea and nausea were of greatest concern.

Conclusion

Pruritus impacts QoL in PBC in several ways and the limitations of existing treatments mean patients would welcome alternative options. Future research will establish the relative importance to patients of treatment aspects such as administration, benefits, and tolerability.
Funding: GSK (218705).
Encore statement: This abstract was originally presented as poster 4550-C at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting on November 13, 2023, this encore is presented on behalf of the original authors with their permission.

Disclosure

HTS, AW and PJFT are employees of, and hold stocks/shares in, GSK. AH is an employee of, and holds stocks/shares in, GSK and is a former employee of, and holds stocks/shares in, Novartis. DS and KM are employees of Evidera who were funded by GSK to carry out this study. JS and SH are employees of Evidera who were funded by GSK to carry out this study and are minority stockholders in Thermofisher Scientific as part of their employment with Evidera.