< Back 2024. The PSC Forum: Characterizing Acute Cholangitis Experiences: Further Results from the PSC Flare or Cholangitis Attack Patient Survey Note: This is a continuation of the research from the 2024 AASLD Liver Meeting Abstract and Poster: 4346: DEFINING ACUTE CHOLANGITIS AS A CLINICAL OUTCOMES ENDPOINT IN ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS: RESULTS OF A MULTINATIONAL PATIENT SURVEY TO DEVELOP A PATIENT-REPORTED OUTCOMES MEASURE. Original Poster Authors: Stephen Rossi, Martine Walmsley, Brian Thorsen, Joanne Hatchett, Donna Evon, Bryce Reeve, Rachel Gomel, Mary Vyas, Ricky Safer, Kerrie Goldsmith, Alastair Garfield, Pamela Vig, Veronica Miller, Michael Trauner Presenter: Brian Thorsen Presentation slides are available here. Previous Next

< Back Pliant Therapeutics: Phase 2a Evaluation of Safety, Tolerability, and Pharmacokinetics of PLN-74809 in Patients With PSC Under construction - check back soon!

< Back Pliant Therapeutics Phase 2a Evaluation of Safety, Tolerability, and Pharmacokinetics of PLN-74809 in Patients With Primary Sclerosing Cholangitis (PSC) Phase 2a Study Previous Next

< Back Escient Pharmaceuticals: PACIFIC Study - EP547 in Subjects With Cholestatic Pruritus 2021 Talyor, K, et al. FIRST-IN-HUMAN STUDY OF EP547, A POTENT AND HIGHLY SELECTIVE MRGPRX4 INVERSE AGONIST IN DEVELOPMENT FOR TREATMENT OF CHOLESTATIC PRURITUS. HEPATOLOGY, VOLUME 74, NUMBER 1 (SUPPL), AASLD Abstracts. Please find Abstract #1272. Text available without subscription at doi/epdf/10.1002/hep.32188. Notes: Results of a phase 1 safety study of EP547.

Learn the basics about primary sclerosing cholangitis, a rare liver disease affecting the bile ducts. About PSC What: Primary Sclerosing Cholangitis (PSC) is a rare liver disease that damages the bile ducts inside and outside the liver. With PSC, bile ducts are inflamed, and the inflammation leads to scarring and narrowing of the affected ducts. Eventually, blockages may occur. As the scarring blocks more and more ducts, bile becomes trapped in the liver. Such blockages damage the liver and can result in fibrosis and cirrhosis of the liver and liver failure. Patients may eventually require a liver transplant. Aside from transplant, there currently are no approved treatments to stop or slow the progression of PSC. PSC often occurs with inflammatory bowel disease (IBD), most often ulcerative colitis (UC) and sometimes Crohn’s disease. It is estimated that more than 75 percent of PSC patients have IBD. PSC also is associated with other autoimmune diseases. Who: PSC can affect all ages and genders, but affects about twice as many males as females. PSC is most common in northern European countries. A reliable epidemiological estimate of the total world population of PSC patients is not well established; however, it is estimated that there are more than 30,000 PSC patients in the United States, 3,000 in Canada, and 10,500 in the UK. Causes: Although no one knows the causes of PSC, exciting research is underway to better understand the disease, develop more effective treatments, and, eventually, find a cure. This registry aims to facilitate and accelerate this research. Visit the Registry Impact page to see Registry participant data at work in research. More Information: Please visit the PSC Partners website at www.pscpartners.org for a comprehensive and trusted source of information about PSC. You will find resources for patient and caregiver support, as well as links to patient support groups outside the US.

< Back 2025. AASLD The Liver Meeting: SYMPTOM PROFILES AMONG ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS IDENTIFIES LOW, MODERATE AND HIGH BURDENED GROUPS Authors: Donna Evon, Kaya Merkler, Chelsea Anderson, Joanne Hatchett, Ricky Safer, Rachel Gomel, Sasha Deutsch-Link, Christopher Bowlus, Bryce Reeve Abstract: Background: Patient-reported symptoms of PSC are not well documented. The study’s aim was to understand the prevalence of PSC symptoms and identify distinct symptom profiles and their association with demographic and clinical characteristics among symptomatic adults. Methods: Adults were recruited through the PSC Partners Patient Registry and PSC hepatologists in the U.S. Individuals were eligible if they were 18 or older, symptomatic in the last year, and not waitlisted or a transplant recipient. Individuals engaged in a phone survey that screened for the presence (yes/no) of 13 PSC-associated symptoms in the last month. Sociodemographic and medical history were self-reported. Latent class analysis (LCA) was used to identify symptom-based participant profiles based on symptom presence and participants were assigned to one symptom class. Multinomial logistic regression models were used to evaluate demographic or clinical factors associated with profile membership. Results: Participants (n = 126) were on average 47 years old (range:20-81), 60% female, 88% white, 8% black and 3% asian, 59% had inflammatory bowel disease (IBD), 39% reported cirrhosis, 75% were taking ursodiol (UCDA), and 57% had a history of cholangitis attacks. The most prevalent symptoms in the last month included fatigue (83%), daytime drowsiness (73%), and liver pain, cognitive impairment, anxiety, sleep disturbance and itch (all 62-63%). The LCA and clinical interpretation suggested a 4-symptom profile solution: (1) “Low Symptom” class (22% of sample), (2) “Moderate Symptom” class (21%), (3) “Moderate+Distressed” class (39% of sample), and a “High Symptom” class (18%). In unadjusted models, compared to the Low Symptom class, females were more likely than males to be in the High Symptom class (OR =4.36; 95% CI: 1.31-14.51), participants taking UCDA were less likely to be in the High Symptom class (OR =0.25; 95% CI: 0.08-0.80) and those with a history of cholangitis attacks were less likely to be in the Moderate Symptom class (OR = 0.22; 95% CI: 0.07-0.70). In the multvariable model, only a history of cholangitis attacks (p = 0.03) was statistically associated with class membership. A statistically significant worsening trend was observed in PROMIS® Global Physical and Mental Health T-scores from the Low to the High Symptom classes. Conclusion: Among symptomatic adults with PSC, over 60% report fatigue, daytime drowsiness, liver pain, cognitive impairment, anxiety, sleep disturbance and itch in the past month. The four-symptom profiles ranging from low to high burden needs validation in larger cohorts as they have clinically meaningful implications for patients’ health status and care of symptomatic individuals. Previous Next

< Back Escient Pharmaceuticals Study to Evaluate EP547 in Subjects With Cholestatic Pruritus Due to Primary Biliary Cholangitis or Primary Sclerosing Cholangitis (PACIFIC) Phase 2 Study Previous Next

< Back 2020 PFDD: Externally-led Patient Focused Drug Development Meeting on PSC with FDA The externally-led PFDD Forum for PSC was organized by PSC Partners, with the FDA, and held in October 2020. In preparation for the PFDD Forum, PSC Partners developed the Our Voices survey in order to systematically capture the frequency, and severity of PSC symptoms and to quantify symptom impact on physical function and daily life. This survey was distributed through the PSC Partners Patient Registry and the broader community from July to September 2020. Visit the PSC Partners website for links to view the meeting on-demand. The Voice of the Patient Report was delivered to the FDA in early 2022 and is available through the FDA for reference by industry and regulators in pursuit of drug development and an approved treatment for PSC. The report is included in the link above.

< Back Mirum Pharmaceuticals A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (VISTAS) Phase 2 Study Previous Next

Help researchers worldwide unlock the mysteries of primary sclerosing cholangitis (PSC). Complete your profile and join PSC Partners Seeking a Cure in advancing PSC research towards a cure. Your participation is important. Welcome to the PSC Partners Patient Registry! If you or a loved one has the rare liver disease primary sclerosing cholangitis (PSC), you are in the right place! This site was developed and is supported by PSC Partners Seeking a Cure, a nonprofit organization dedicated to providing education and support to PSC patients, families, and caregivers. Another vital mission of PSC Partners is to support researchers in their quest to find causes, treatments, and cures for this rare disease. The PSC Partners Patient Registry was initiated in 2014 in response to the difficulty of locating PSC patients and collecting PSC data to conduct research on our rare disease. Our aim with the Registry has been to add the patient’s voice to every facet of PSC research and to facilitate and speed up PSC research towards a cure. All those diagnosed with PSC are encouraged to participate. Parents can join the Registry to enroll their child. A spouse can participate on behalf of their partner. All data in the Patient Registry is de-identified. When a researcher requests information, no identifying information (name, date of birth, address, or phone number) ever leaves the Registry. Researchers, approved by the PSC Partners Patient Registry Team, request de-identified registry data for their research, for patient recruitment in clinical trials, and for developing clinical study protocols. Participants of the PSC Partners Patient Registry represent an important voice of the PSC community: They engage in research by completing surveys and joining clinical trials. They help us advocate by expressing their unmet needs. When new treatments arrive, the Registry can help track the effectiveness of the new drug and can speed up drug approval. If you or your child appears to be a match for a study or a clinical trial, the registry coordinator will contact you. It would be your choice to connect with the researcher(s) to let them know if you are interested. To join, we request that you enter your General Information, give consent, complete the Clinical Survey, and that you provide a proof of diagnosis which could be a recent or old MRCP or ERCP result, or a doctor’s report that states you have PSC. You can learn more about the specifics of the registry by clicking on the various links on this site. We hope you will be inspired to join the PSC Partners Patient Registry. Your voice is crucial for this small and dispersed PSC community! You can help researchers worldwide by taking around 30 minutes of your time to complete your profile - or your child’s or loved one’s profile. Join the hundreds of men, women, and children who are helping to advance PSC research towards treatments and a cure! We are together in this fight, whatever it takes! Join us! The registry coordinator is available to respond to any questions you may have. Please don't hesitate to reach out by email at registrycoordinator@pscpartners.org . Brian Thorsen, Registry Director Fernanda Quevedo, Registry Assistant Director Sharon Nanz, Registry Coordinator Chris Bowlus, MD, Registry Scientific Advisor , Chief of Gastroenterology, UC Davis All rights reserved © PSC Partners Seeking a Cure

< Back 2022 Participation in multinational survey on pruritus in PSC. Results presented at International Liver Congress. Authors: Kris Kowdley, et al. Mirum Pharmaceuticals Presents New Data at The International Liver Congress™ of the European Association for the Study of the Liver (EASL) Jun 24, 2022. Largest multinational survey highlights impact of pruritus on quality of life for patients with primary sclerosing cholangitis. “We are pleased to share new data at the 2022 EASL congress,” said Dr. Pam Vig, head of research and development at Mirum. “The large multinational PSC survey shows that adults with PSC often experience significant and debilitating pruritus which impacts their day-to-day quality of life, despite use of off-label antipruritic medications. Thus, there remains an urgency for a treatment to address this most burdensome symptom, and we are excited to be evaluating the potential of volixibat, a minimally absorbed IBAT inhibitor, in patients with PSC.” Poster 756 : Impact of pruritus in primary sclerosing cholangitis (PSC): a multinational survey By Dr. Kris Kowdley, et al. The multinational survey, conducted by Hetz Israel, PSC Australia, PSC Partners (through the PSC Partners Patient Registry), and PSC Support, assessed results from a collection of patient-reported outcomes from people living with PSC (n=482). The aim of the survey was to assess the presentation and severity of cholestatic pruritus and its broader burden on patients with this rare liver disease. The 39-question survey captured the age and gender of respondents as well as whether they also have inflammatory bowel disease. In addition to pruritus, inflammatory bowel disease and chronic liver disease associated with PSC can negatively impact the quality of life and can lead to depression and anxiety. The survey results showed that: Of the 91% of patients who reported experiencing itch following their diagnosis, 46.4% of respondents experienced >15 episodes (an episode is defined as a prolonged period of itching for at least 7 days). Respondents were asked to rate the worst itch experienced in the last 24 hours, and the median worst itch was 6 out of 10 in those who responded (scale from 0-10). Their previous itching episode was reported as 8 out of 10. 31% of patients who reported having a current itching episode stated that they have been continuously itching for >12 months. The survey assessed the overall impact of pruritus on respondents’ lives with responses indicating: Itch was worst in the evening or nighttime (96%) Mood changes including but not limited to anxiety, irritability, and feelings of hopelessness (58%) Itch led to disruption of day-to-day activities (50%) Itch lasted ≥1 month in duration (45%) Disruption of daily responsibilities due to itch that lasted >30 days (32%), and reported it lasting >12 months (18%) Missing school or work (22%) Other findings showed that half of respondents (235/482) reported using ≥2 medications but 75% (177/235) described only partial or no relief with the interventions. Data from the survey concluded that pruritus related to PSC has a major adverse impact on quality of life (e.g., sleep, mood, fatigue), and interferes with daily activities in a substantial proportion of patients, yet it remains inadequately treated in most patients. There is a high unmet need for the development of safe and effective therapies to treat PSC.

< Back 2025. AASLD The Liver Meeting Abstract and Poster: 4476: IDENTIFYING GAPS IN PSC PATIENT EDUCATION AND UNDERSTANDING OF CHOLANGIOCARCINOMA SCREENING AND SURVEILLANCE PRACTICES: RESULTS OF A MULTINATIONAL PATIENT SURVEY Authors: Brian T. Thorsen, Martine Walmsley, Susan O’Dell, Rachel Gomel, Mark Chatterley, Mary P. Vyas, Stephen J. Rossi Abstract: Background and Aims: People with primary sclerosing cholangitis (PSC) have up to a 20% lifetime risk of cholangiocarcinoma (CCA). CCA surveillance is challenged by the poor performance of current screening methods. We report results of a multinational patient survey to assess knowledge of CCA surveillance and patient-provider communication on CCA risk and surveillance results. The primary goal was to identify gaps in knowledge and communication in anticipation of new CCA screening tests. Methods: A survey for adults with PSC was developed by PSC Partners and PSC Support patient organizations. Patients reported relevant medical history, CCA surveillance tests performed (MRCP, CA 19-9, ERCP), risk and results discussion, and CCA education. The data was analyzed to compare rates of proactive CCA discussion from providers by patient characteristics, region, and provider type. Results: A total of 623 responses met screening criteria, primarily from the US (45%) and UK (31%). 31 patients reported a biliary tract cancer diagnosis (25 CCA, 6 GBC). Only 51% of patients reported proactive discussions by their provider of CCA risks and surveillance. Among patients who had a proactive CCA discussion, 56% reported having sufficient information regarding CCA, versus only 15% among those without proactive discussion ( p < 0.001). Patients in North America reported a higher rate of proactive discussion than Europe (58.2% / 36.9%, p < 0.001), and rates were higher for hepatologists than gastroenterologists (55.0% / 38.3%, p < 0.001). Discussion rates were not significantly different across patient gender, age, and time since PSC diagnosis. Annual MRCP surveillance was reported by 68% of patients, with significant differences by region and provider type. Proactive CCA discussion was strongly associated with more frequent use of annual MRCP. Regular CA 19-9 testing was reported by 36% of respondents. MRCP and CA 19-9 testing results for CCA surveillance were discussed with 27% and 72% of patients who had those tests, respectively. Only 36% of patients felt they had adequate CCA information; the majority seeking information from online search, followed by patient organizations and their care team. Patients consistently reported significant anxiety and fear regarding CCA risk and the need for a reliable, non-invasive screening test. Conclusion: The results show high variability in discussion of CCA risk and test results and CCA surveillance practice in a population burdened with anxiety and fear around CCA. These results are influenced by differences in regional guidelines, healthcare systems, and the lack of highly predictive non-invasive diagnostic and prognostic tools. Promising novel biomarkers to detect early CCA and risk will impact the current routine surveillance practices and patient education needs. Appropriate global patient/provider education and communication programs should be implemented to support the use and delivery of such tests. Previous Next