< Back 2025. Digestive Disease Week Abstract and Poster: DIVERSITY AND SOCIOECONOMIC STATUS OF PATIENTS WITH PRIMARY SCLEROSING CHOLANGITIS (PSC) AND ITS IMPACT ON HEALTHCARE RECEIVED, A SURVEY OF PSC PARTNERS PATIENT REGISTRY Authors: Kidist Yimam, MD, Vishwajit Kode, MD, Rachel Gomel, Mary P. Vyas, Joanne Hatchett, Brian Thorsen, Willie McKinney, PhD, Ricky Safer Abstract: (Updated results from a larger response pool are presented in the poster above.) Background: Primary sclerosing cholangitis (PSC) is a rare, cholestatic liver disease, characterized by progressive stricturing of bile ducts, with risk of progression to cirrhosis and liver failure needing liver transplantation. There is no approved medical therapy for PSC except liver transplantation. A timely diagnosis, clinical monitoring and access to a liver center with opportunities to participate in PSC clinical trials is crucial in the care of patients with PSC. Aims: Evaluate the association between socioeconomic status and patients’ diversity on healthcare experience and clinical outcomes in patients with PSC. Method: A detailed, 20-point, health-equity survey was developed by PSC Partners Health Equity Working Group. The survey included questions on demographic data, race, ethnicity, sexual orientation, location of residence, health insurance type, educational level, household size and income, baseline clinical data and outcomes. The survey was administered via the PSC Partners Patient Registry. The answers were summarized, and statistical analysis was performed for potential associations between diversity and socioeconomical status and clinical care and outcomes. Results : 421 patients completed the health equity survey in the Registry. Ten patients were excluded due to missing crucial data. Their baseline characteristics with clinical outcomes (Table 1) and healthcare access and experiences (Table 2) are summarized below. The Registry has mostly White patients with English language preference and female predominance. Most patients are not employed fulltime (56%) and no dependents (48%). One third of patients experienced delay in PSC diagnosis. Most patients have primary care doctors, gastroenterologist and hepatologist (all). 65% of patients are not participating in PSC clinical trials. In this very active patient advocacy group with 33% having themselves or family member in the healthcare field, only 65 % have a good understanding of their PSC management. Significant findings include: IBD diagnosis predicting colonoscopy adherence (p-values <0.0001, liver transplant status associated with better understanding of PSC management (p=value <0.001) and household income correlating with satisfaction of care (p-value <0.001). Conclusion: This health equity survey in PSC highlighted the need to make a robust effort to include diverse patients in the PSC registry. Additionally, efforts need to be made to educate patients and caregivers about PSC as we anticipate that the understanding of the disease and engagement in PSC clinical trials is even lower in PSC patients not connected with PSC Partners. We plan to expand this healthy equity survey to the general PSC population globally to understand healthcare disparities and their causes so that meaningful efforts can be made to mitigate these variables, promoting health equity for all affected by PSC. Previous Next

< Back European Joint Program on Rare Diseases and national funding agencies in Germany, Canada, Belgium, Poland and Hungary Improving health-related quality of life in patients with rare autoimmune liver diseases by structured peer-delivered support: a transnational effectiveness-implementation hybrid trial Previous Next

< Back 2023. AASLD Liver Meeting Abstract and Poster: 4558-C: NETWORK ANALYSIS OF SYMPTOMS EXPERIENCED BY PATIENTS WITH PRIMARY SCLEROSING CHOLANGITIS Authors: Melinda Wang, Amy M. Shui, Joanne Hatchett, Ruth-Anne Pai, Rachel Gomel, Mary Pressley Vyas, Sarah Curup Callif, Ricky Safer, Christopher L. Bowlus, Donna M. Evon, Jennifer C. Lai, Michael Li. Journal: Hepatology. 78(S1):S1-S2154, October 2023. Abstract: Background: Patients with primary sclerosing cholangitis (PSC) experience numerous symptoms that impact quality of life. PSC Partners Seeking a Cure developed the international Our Voices survey to capture the presence, severity, and impact of PSC symptoms on quality of life. We sought to identify PSC-specific symptom clusters and relationships between these symptoms to better understand the lived experience of PSC patients. Methods: Presence and severity of 16 symptoms potentially related to PSC were identified from the Our Voices survey. LASSO regularized partial correlation network analyses were performed to model unique interactions between symptoms. The primary analysis utilized symptoms reported within the past 6 months of survey completion. Secondary analyses included active symptoms and symptoms at their worst. Expected influence, which accounts for both positive and negative associations and assesses the nature and strength of a node’s cumulative influence within a network, was computed for all symptoms in each network. The Spinglass algorithm was employed to identify symptom communities. Results: Among 970 total participants, 54% were women, most were white (70%), and 70% had inflammatory bowel disease (IBD). 88% of patients reported experiencing symptoms. Four dominant communities were identified across networks including psychological symptoms (anxiety, depression), gastrointestinal symptoms (anorexia, nausea/vomiting, liver pain, abdominal pain), cholestasis-related symptoms (pruritus, insomnia, night sweats, jaundice), and global symptoms (fatigue, weakness, joint pain, other pain, brain fog, encephalopathy) (Figure). Of the 16 PSC symptoms, fatigue and weakness consistently had the greatest expected influence across the multiple networks generated in the primary and secondary analyses. On subgroup analysis, symptoms with the strongest influence among patients with and without IBD were similar (IBD: fatigue and insomnia; no IBD: fatigue and anorexia). Conclusion: Our analyses suggest that the complex relationships between PSC symptoms organize into four dominant, clinically-relevant clusters. Fatigue and weakness emerged as the symptoms with the strongest influence across the primary and secondary analyses. Our data emphasize the co-occurring nature of PSC symptoms, identify highly influential symptoms, and highlight the need to develop patient-reported outcome measures to assess changes in these key symptoms in clinical trials Previous Next

< Back 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. 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 Abstract: Background: Acute cholangitis (AC) is a clinically significant, poorly understood PSC complication challenged by the lack of standardized diagnostic and treatment criteria. We report the results of a multi-national patient survey to characterize the patient experience during AC as a first phase to develop a patient-centered PROM for use in clinical trials and patient care. Methods: A detailed anonymized patient survey was developed by PSC Partners and PSC Support patient organizations. Adults diagnosed with PSC and ≥1 cholangitis attack were invited to complete the survey. Relevant medical history, AC symptom frequency and severity (before, during and after AC), and medical interventions were collected. A visual abdominal pain locator was used to further characterize AC pain location. Results: A total of 355 of 428 responses met screening criteria. Timing of first AC attack relative to PSC diagnosis ranged from before (38%), concurrent (14%) and after (48%). Over 65% had AC in the past year, with 45% having ≥2 attacks. Fatigue (92%) and liver pain (86%) were reported most frequently, with fatigue (30%) and fever/chills (24%) having highest severity during AC (Fig 1A). Severity improved for all symptoms after AC, except anxiety. Symptom improvement was greatest with fever/chills (55%), anorexia (51%) and liver pain (51%). Complete symptom resolution was highest for vomiting (50%), fever/chills (46%), night sweats (31%) and nausea (29%), while lowest with fatigue (6%). More patients reported no change in symptom severity after AC, with the least change seen with jaundice, brain fog and pruritus. Anxiety worsened the most after AC at 12% while fatigue was the most reported new-onset symptom at 33%. Abdominal pain was largely located in the right upper quadrant (RUQ) during AC, independent of IBD, cirrhosis or transplant (Fig 1B). Other reported pain locations >10% were body (73%), back (17%) and shoulder (10%). The majority of IBD patients (70%) clearly differentiated AC symptoms from IBD flares. Medical care was sought by 76%, of which 86% took oral and/or IV antibiotics. Other procedures included MRI (68%), ultrasound (57%), ERCP (44%) and stent/balloon dilation (26%). Despite AC treatment and/or resolution, 32% did not return to their perceived baseline health status, reporting persistent or new onset fatigue (44%), RUQ pain (15%) and brain fog (11%). Conclusion: This detailed survey demonstrates a broad dynamic range of the frequency and severity of symptoms over the clinical course of AC. Symptom improvement is seen in a subset of patients, but the majority show no improvement or worsening of symptoms despite treatment or attack resolution. Fatigue and anxiety are under-recognized symptoms reported with AC and warrant further study. No single symptom provides adequate specificity or sensitivity for AC diagnosis or monitoring, clearly supporting the significant need to develop a multi-symptom PROM for clinical trials and patient care. Previous Next

< Back National Institutes of Health: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach Previous Next

< Back 2024. Caregiver-reported symptom burden and preferences for therapeutic goals in pediatric primary sclerosing cholangitis Authors: Holly Payton Shifman , Joanne Hatchett , Ruth-Anne Pai , Ricky Safer , Rachel Gomel , Mary Vyas , Michael Li , Jennifer C. Lai , Sharad I. Wadhwani Journal: Journal of Pediatric Gastroenterology and Nutrition. 20 June 2024. Article Link Abstract: This study analyzed qualitative and quantitative survey responses from 51 pediatric primary sclerosing cholangitis (PSC) patients and caregivers using the PSC Partners Patient Registry– Our Voices survey. The most common symptoms reported by children/caregivers include: fatigue (71%), abdominal pain (69%), anxiety (59%), appetite loss (51%), insomnia (49%), and pruritus (45%). When experiencing symptoms at their worst, over half of patients/caregivers reported limitations in physically demanding activities (67%), work/school duties (63%), social life activities (55%), and activities for fun or exercise (53%). Over half of patients/caregivers expressed willingness to participate in clinical trials, however none reported ever participating in trials for new or investigational PSC drugs. This study revealed a substantial patient/caregiver-reported symptom burden for children with PSC that impacts quality of life and limits access to clinical trials. Future efforts should focus on developing patient-centered clinical endpoints for PSC trials, increasing trial availability for pediatric PSC patients, and reducing logistical barriers to trial involvement. Previous Next

< Back 2015 Trial Design and Endpoints for Clinical Trials in Adults and Children with Primary Sclerosing Cholangitis: GREAT Workshop, AASLD-FDA Collaborative. Presentation of Registry Participants Survey Responses Ricky Safer, CEO of PSC Partners Seeking a Cure, presented PSC Partners Patient Registry participants responses to a survey on patient attitudes to and concerns about participating in clinical trials at “Trial Design and Endpoints for Clinical Trials in Adults and Children with Primary Sclerosing Cholangitis: GREAT Workshop,” AASLD-FDA collaborative workshop, Bethesda, MD, USA March 3-4, 2015.

< Back Chemomab: SPRING Study - CM-101 in PSC Patients 2024 Snir, T et al. Machine Learning Identifies Key Proteins in Primary Sclerosing Cholangitis Progression and Links High CCL24 to Cirrhosis. https://www.mdpi.com/1422-0067/25/11/6042 2024 Greenman, R et al. The Role of CCL24 in Primary Sclerosing Cholangitis: Bridging Patient Serum Proteomics to Preclinical Data. https://www.mdpi.com/2073-4409/13/3/209

< Back 2016. Medical Treatments in Patients with Primary Sclerosing Cholangitis Differ Between Europe and North America Poster and abstract presented at EASL International Liver Congress 2016 Authors: Gomel, Rachel & Safer, R. & Lindor, Keith & Everson, Gregory & Bowlus, C Journal: Journal of Hepatology. 64. S641. 10.1016/S0168-8278(16)01194-6. https://doi.org/10.1016/S0168-8278(16)01194-6 Excerpts: "Conclusions: Treatment of PSC and concomitant IBD differs between Europe and NA and is not accounted for by differences in disease characteristics. Medication differences may need to be considered in studies of disease progression." Data from PSC Partners Patient Registry Previous Next

< Back 2015. Characterization Of A Patient Driven International Registry For Primary Sclerosing Cholangitis AASLD The Liver Meeting Poster #620 Authors: Rachel Gomel, Ricky Safer, Jesse A. King, Estella M. Geraghty, Gregory T. Everson, Keith D. Lindor, Christopher L. Bowlus Journal: AASLD Hepatology Posters (Abstracts 289–2348)Poster Sessions First published:01 October 2015 https://doi.org/10.1002/hep.28162 https://doi.org/10.1002/hep.28162 Excerpts: "SUMMARY Participants in the PSC Partners Registry have characteristics that are similar to those of other cohors with the exception of a lower male prevalence. While pain and pruritis are common in PSC patients, we observed a wide spectrum of severity of these symptoms among participants in the registry. Over 50% of participants are taking UDCA. CONCLUSION The PSC Partners Patient Registry represents a unique resource for future research and can inform clinical trial design." Data from PSC Partners Patient Registry Previous Next

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News and Announcements Join our Email List! Choose a category: Jul 29 Chemomab Announces CM-101 Phase 2 SPRING Trial Results Three Recent Announcements from: Pliant, NGM, and Chemomab PSC Partners is encouraged by the initial positive data from the CM-101 Phase... Jul 22 Positive Update on Pliant’s INTEGRIS-PSC Phase 2a Trial On July 15, 2024, Pliant Therapeutics announced positive results from their Phase 2a INTEGRIS-PSC trial for bexotegrast in treatment of... Jun 24 New Article on an Adaptive Treatment Strategy for Use of Vancomycin in PSC The repurposing of approved medications with potential new efficacy in rare diseases is challenging, as these medications are unlikely to... Jun 21 Expanded Inclusion Criteria for Mirum’s VISTAS PSC Study for Pruritus Treatment The following is a message from Mirum Pharmaceuticals regarding their VISTAS study for volixibat, a possible new treatment for... Mar 18 A New Era for the PSC Partners Registry Community Dear Registry Participants (Current and Future), This year is the 10th anniversary of the PSC Partners Patient Registry, a perfect time... Sep 30, 2023 Preliminary Results from INTEGRIS-PSC Phase 2a Clinical Trial Exciting news about the Pliant Therapeutics PSC clinical trial preliminary results from their global INTEGRIS-PSC Phase 2a trial for... Jul 14, 2023 Health Equity in PSC Survey Health equity is the state in which everyone has a fair and just opportunity to attain their highest level of health*. The mission of PSC... Jun 30, 2023 The VIADUCT Newsletter Issue 6 Issue 6 - June 2023 In this issue: From the Registry Director • New CZI Grant Awarded to PSC Partners • Patient Focus Groups Provide... Jun 24, 2023 PRIMIS (Phase 3 Study of Cilofexor) News from EASL Congress 2023 News from the EASLCongress 2023 meeting in Vienna From the "late-breaking" abstract session on June 24, 2023, results of the pre-planned... Feb 27, 2023 Oral Vancomycin and Insurance Coverage Survey Results: Oral Vancomycin and Insurance Coverage PSC Partners received a request from patients to survey the PSC patient community... Oct 30, 2022 The VIADUCT Newsletter Issue 5 Issue 5 - October 2022 Welcome! In this issue: • From the Registry Director • The Clinical Trial Landscape in PSC • Current International... Sep 28, 2022 PRIMIS (Phase 3 Study of Cilofexor) News Statement From Gilead Sciences on September 27, 2022 Early Termination of Gilead-Sponsored Study GS-US-428-4194: A Phase 3, Randomized,... May 25, 2022 The VIADUCT Newsletter Issue 4 In this issue: • From the Registry Director • Mini-PFDDs • Patient Registry Video Tour • Help Us Grow & Diversify the Registry! • PSC... Dec 1, 2021 The VIADUCT Newsletter Issue 3 Welcome! In this issue: • From the Registry Director • Profile: Dr. Frank Anania of the FDA • Your Registry Data at Work • PSC Partners... Aug 24, 2021 The VIADUCT Newsletter Issue 2 Dear Registry Participant, Thank you for your participation in the PSC Partners Patient Registry. We are happy to bring the second issue... Mar 1, 2021 Proof of Concept Phase 2 Trial of IMU-838 with Early Positive Results A proof-of-concept Phase 2 trial conducted by Mayo Clinic and sponsored by Immunic has shown very early positive results. We hope that... Jan 28, 2021 Clinical Trials Updated: PLN 74809 and Volixibat The clinical trials resources page has been updated on 1/27/21 to reflect two clinical trials currently recruiting. PLN 74809: Phase 2a... Nov 13, 2020 Successful registry appeal garners strong support On October 6, 2020, the PSC Partners Registry team sent out an email alert regarding a Harvard Brigham and Women’s Hospital nutrition... Sep 23, 2020 Nutrition Study Recruiting Now (US residents) Check out this video invitation from PSC nutrition study Principal Investigator Dr. Josh Korzenik to participate in a diet and nutrition... Aug 22, 2020 Ask the PSC Expert - Dr. Gideon Hirschfield Joanne Hatchett, PSC Partners Medical Science Liaison, sat down for a Zoom interview with Dr. Gideon Hirschfield. Dr. Hirschfield is the... All Posts The VIADUCT Newsletter Clinical Trials