< 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

< Back 2020 Diet in PSC Study (DINER) - Active Now The PSC Collaborative at the Brigham and Women’s Hospital is recruiting patients with PSC for a pilot research study to investigate the role of diet and nutrition in the development and treatment of PSC. The study will evaluate the feasibility of two different dietary interventions – the low sulfur/vegan diet versus the specific carbohydrate diet. Researchers believe that altering diet may have an impact on the disease by altering the microbiome, bile salts, fatty acids, immune function or other factors. The samples and information we collect in this study may advance other research to help individuals with PSC worldwide. For more information visit here.

< Back University of Minnesota Evaluation of an Oral Microbiota-based Therapeutic as a Treatment Option for PSC Early Phase 1 Study Previous Next

PSC Registry Overview for Researchers At a Glance 2,776 Participants from 55 countries 6,500 Completed surveys 23 Publications made possible through Registry data and recruitment as of Jan 2026 Research Partnerships DATA SHARING Aggregate or de-identified individual-level Registry data can be provided for approved research. Additional questionnaires can be sent to participants if needed. STUDY RECRUITMENT The Registry emails info on clinical trials and other studies to patients matching the criteria. For confidentiality, it is up to the patient to contact the investigators. For more details, read the Registry's Data Access Policy . CONTACT US Patient Voices in Action Source: Stephen Rossi et al. "Defining Acute Cholangitis as a Clinical Outcomes Endpoint in Adults with Primary Sclerosing Cholangitis" Source: Michael Li et al. "The Externally-Led Patient-Focused Drug Development Meeting for Primary Sclerosing Cholangitis: Insights on Patient Involvement in Clinical Trials" Current Registry Surveys To date, de-identified survey data has been used in 19 publications. Links and more information can be found on the Registry Impact page. Clinical Survey: This annual survey collects core medical information, family history, current symptoms, medications, and more. With 11 years of data, and this provides a rich data set for analysis, and is used to pre-screen for clinical trial recruitment messages. Health Equity in PSC: This survey collects additional demographic data and information about access to medical care. It also measures patients' confidence in and satisfaction with their care. Acute Cholangitis: Patient history, symptom course, and care received during most recent flare/cholangitis episode are collected in this survey. PSC & Cholangiocarcinoma (CCA): This survey elicits patient experience surrounding monitoring for CCA, awareness of and education about CCA, and discussion with their clinicians about CCA. Symptom Assessment: This survey is part of the Symptom Assessment Project, a PSC Partners initiative to develop fatigue, cognitive impairment, and liver pain PRO measures for potential use as clinical trial endpoints. The goal of this survey is to support validation of the PRO instruments in a real-world cohort. Past surveys include the Our Voices Survey, the results of which were presented to the FDA at the Patient-Focused Drug Development Forum for PSC; the PSC-specific COVID-19 Survey; the Oral Vancomycin and Insurance Survey, which helped inform a legislative advocacy effort; and the external RAND Health SF-36 quality-of-life measure. Clinicians help the Registry grow! Patients often learn of the Registry through their doctor. It is an easy and secure way for all with PSC to help advance PSC research, whether pre- or post-transplant. Caregivers can join on behalf of children or adults with PSC (adults must consent). We encourage you to discuss the Registry with your patients. Informational materials including brochures, flyers, and posters are available upon request.

< Back 2021. FINDINGS FROM THE PATIENT-FOCUSED DRUG DEVELOPMENT FORUM ON PRIMARY SCLEROSING CHOLANGITIS AASLD The Liver Meeting Poster #630 Conclusions: Findings from the Our Voices survey informed the development of the externally-led PFDD Forum for PSC and highlight the need for a more robust, validated Patient Reported Outcome Measure (PROM) for PSC. Overall, our results demonstrate the willingness of the PSC patient community to share experiences and guide the development of PROMs, which requires further perspective and expertise from the PSC research community. Previous Next