< Back 2019. Clinical Characteristics and Outcomes Reported by Patients with Primary Sclerosing Cholangitis Through an Online Registry Authors: Kuo A, Gomel R, Safer R, Lindor KD, Everson GT, Bowlus CL. Journal: Clin Gastroenterol Hepatol. 2019;17(7):1372‐1378.doi:10.1016/j.cgh.2018.04.047 Excerpts: "We analyzed data from the worldwide PSC Partners Patient Registry, an international online database established in 2014 to obtain information from individuals with PSC or their caretakers and compare symptoms, disease progression, and treatments of PSC in the United States and other countries." "Conclusions: Our findings from an analysis of data from the PSC Partners Patient Registry confirm those from previous studies, although we found a higher proportion of individuals with PSC to be female. In addition to allowing efficient collection of patient-reported outcomes, the patient-driven registry allows for inclusion of previously under-represented cases of PSC." Pubmed.gov listing and abstract here. Full-text article available here. Previous Next

< Back 2018 Presentation on PSC Databases at the PSC Forum Meeting 2 Presentation by Rachel Gomel, Registry Director at the PSC Forum April 10, 2018 in Paris, France, documenting the status of PSC databases and registries around the world. Presentation slides available here . PSC DATABASES/ REGISTRIESPSC DATABASES/ REGISTRIES The PSC Patient Databases Working Group requested that the Forum for Collaborative Research create a registry webpage to highlight the various PSC databases currently available. Overall, the goal of this webpage is to sensitize and inform PSC patients about specific registries that are available for them to join and to encourage researchers to engage in data-sharing through collaboration.

< Back 2019. Pregnancy Experiences in Women with Primary Sclerosing Cholangitis AASLD The Liver Meeting Poster #1280 Authors: Dhruv Patel, Chung Liu, Veesta Falahati, Alanna Dubrovsky, Monika Sarkar, Christopher Bowlus Journal: AASLD Hepatology Posters (Abstracts 289–2348) First published:01 October 2019 https://doi.org/10.1002/hep.30941 Excerpts: "Although frequently affecting reproductive-aged women, the impact of PSC and concomitant inflammatory bowel disease (IBD) on pregnancy, is not well-studied. The aim of this survey was to evaluate maternal-fetal outcomes among women with PSC with and without concurrent IBD." Data from PSC Partners Patient Registry Abstract can be found here . Click on the picture below to view as a PDF. Previous Next

< Back Mayo Clinic: Oral Vancomycin for PSC Last updated October 2024. There are a number of publications related to the use of oral vancomycin in PSC. As you review the literature, please remember to critically evaluate the strengths and limitations of each publication. Publications are listed in reverse chronological order within each section. This list is by no means exhaustive, and we have excluded publications about animal studies. Notes are added solely to assist in navigating the list. Readers are encouraged to read the full text to consider the full context of the evidence in the publication. Please see the PSC Partners Statement about the Use of Medications Not Approved by the FDA for PSC . Randomized Clinical Trials 2016 - S Rahimpour et al. “A Triple Blinded, Randomized, Placebo-Controlled Clinical Trial to Evaluate the Efficacy and Safety of Oral Vancomycin in Primary Sclerosing Cholangitis: a Pilot Study.” Journal of gastrointestinal and liver diseases: JGLD vol. 25,4: 457-464. Full text available without paid subscription at doi: 10.15403/jgld.2014.1121.254.rah . Notes: A blinded, randomized, and placebo-controlled 12-week study with 29 adult PSC patients randomized into two groups. Patient symptoms were tracked in addition to biochemistry. Details of trial can be found on clinicaltrials.gov:NCT02605213 . 2013 - JH Tabibian et al. “Randomised clinical trial: vancomycin or metronidazole in patients with primary sclerosing cholangitis - a pilot study. ”Alimentary pharmacology & therapeutics vol. 37,6: 604-12. Full text available without paid subscrition at doi: 10.1111/apt.12232 Notes: A randomised 12-week study with 35 adult PSC patients, no placebo, 4 groups dosed with high or low doses of antibiotics oral vancomycin or metronidazole. Details of trial can be found on clinicaltrials.gov:NCT01085760 . 2013 - JCM Paterson and J F Dillon. “Commentary: vancomycin or metronidazole in patients with primary sclerosing cholangitis?” Alimentary pharmacology & therapeutics vol. 37,9: 915. Full text available without paid subscrition at doi:10.1111/apt.12282 Notes: Commentary on Tabibian et al 2013 article. Prospective Studies 2023 - Quraishi et al. "Oral vancomycin induces remission in PSC-IBD, which is associated with a reduction in bile salt hydrolase and colonic amine oxidase activity" S124, page 2123, Journal of Hepatology 2023 vol. 78(S1) | S100–S1212. Abstract book can be downloaded here . Trial was registered at clinicaltrials.gov (NCT05376228 ). Notes: Phase 2a clinical trial, 15 adults, with goal of identifying "key mechanistic pathways associated with induction of treatment response." 2020 - AH Ali et al. “Open-label prospective therapeutic clinical trials: oral vancomycin in children and adults with primary sclerosing cholangitis.” Scandinavian journal of gastroenterology vol. 55,8: 941-950. Abstract and summarized results can be found at doi: 10.1080/00365521.2020.1787501 . Trial details can be found at clinicaltrial.gov NCT01802073 and NCT01322386 . Notes: Open-label prospective trial of oral vancomycin in 45 children and 14 adults with PSC followed for at least 12 months. Results reported for biochemistry (blood tests), some biopsies, MRCPs and MREs. 2011 - D Pratt et al "An Open Label Pilot Trial of Oral Vancomycin in Primary Sclerosing Cholangitis - Results of the VIP Trial" Gastroenterology, May;140(5). Full text available without subscription at DOI10.1016/S0016-5085(11)63765-7 . Notes: Published as meeting abstract. 7 adult PSC-IBD patients completed 52 weeks of oral vancomycin therapy with pre- and post-therapy biopsy and MRCP. Large Retrospective Study 2024 - A Ricciuto et al. "Oral vancomycin is associated with improved inflammatory bowel disease clinical outcomes in primary sclerosing cholangitis-associated inflammatory bowel disease (PSC-IBD): A matched analysis from the Paediatric PSC Consortium." Alimentary Pharmacology & Therapeutics vol. 59,10: 1236-1247. doi.org/10.1111/apt.17936 Notes: Retrospective matched study of 70 vancomycin-treated and 210 untreated pediatric PSC-IBD patients. Evaluates odds of IBD clinical remission after 1 year of vancomycin. 2021 - MR Deneau et al. “Oral Vancomycin, Ursodeoxycholic Acid, or No Therapy for Pediatric Primary Sclerosing Cholangitis: A Matched Analysis.” Hepatology (Baltimore, Md.) vol. 73,3: 1061-1073. Full text available without subscription at doi:10.1002/hep.31560 Notes: Retrospective, propensity matched analysis of 264 pediatric PSC patients who received UDCA, oral vancomycin, or no treatment. Evaluates surrogate outcomes at 1 year and if transplanted at 5 years. 2021 - DN Assis and C Levy. “Oral Vancomycin or Ursodeoxycholic Acid for Pediatric Primary Sclerosing Cholangitis? The Uncontroversial Need for Randomized Controlled Trials.” Hepatology (Baltimore, Md.) vol. 73,3: 887-889. Full text available without subscription at doi:10.1002/hep.31702 Notes: Commentary on Deneau et al 2021 study. 2021 - KM Johnson et al. "Letter to the Editor: Oral Vancomycin Versus No Therapy for Pediatric Primary Sclerosing Cholangitis" Hepatology (Baltimore, Md.) vol 74,3: 1716-1717. https://doi.org/10.1002/hep.31764 Notes: Notes: Commentary on Deneau et al 2021 study. Review Articles and Meta Analyses 2024 - N Arbabzada, L Dennett, G Meng, and F Peerani. "The Effectiveness of Oral Vancomycin on Inflammatory Bowel Disease in Patients with Primary Sclerosing Cholangitis: A Systematic Review." Inflammatory Bowel Diseases , izae257, 2024. Full text available without subscription at https://doi.org/10.1093/ibd/izae257 Notes: systematic review summarizing and evaluating 9 case reports, 7 case series, 3 cohort studies, and 1 randomized clinical trial. 2019 - A Shah et al. “Effects of Antibiotic Therapy in Primary Sclerosing Cholangitis with and without Inflammatory Bowel Disease: A Systematic Review and Meta-Analysis.” Seminars in liver disease vol. 39,4: 432-441. Full text available without subscription at doi:10.1055/s-0039-1688501 Notes: systematic review and meta-analysis to assess the effect of antibiotic therapy in PSC. 5 studies including 124 PSC patients who received antibiotics were included. 2018 - JL Dammanet al. “Review article: the evidence that vancomycin is a therapeutic option for primary sclerosing cholangitis.” Alimentary pharmacology & therapeutics vol. 47,7: 886-895. Full text available without subscription at doi:10.1111/apt.14540 Notes: 2018 review article summarizing 2 randomized clinical trials, 3 case series, and 2 case reports that together include uncontrolled data from at least 98 patients. 2018 - RW Chapman “Editorial: vancomycin - a promising option for the treatment of primary sclerosing cholangitis?” Alimentary Pharmacology & Therapeutics vol. 47,9: 1321-1322. doi:10.1111/apt.14586 Notes: Editorial response to 2018 Damman et al review article. 2017 - F Saffioti et al. “Pharmacological interventions for primary sclerosing cholangitis: an attempted network meta-analysis.” The Cochrane database of systematic reviews, vol. 3,3 CD011343. 28 March, doi:10.1002/14651858.CD011343.pub2 Notes: a network meta‐analysis of 22 trials including the 2016 Rahimpour et al and the 2013 Tabibian et al RCTs that included oral vancomycin therapy. Case Series and Case Studies 2024 - Sohal, Aalam, and Kris V Kowdley. “Complete Biochemical Remission With Oral Vancomycin in a Patient With Primary Sclerosing Cholangitis and High Serum Immunoglobulin G4 Levels.” ACG case reports journal vol. 11,1 e01256. 16 Jan. 2024, doi:10.14309/crj.0000000000001256 2022 - A Shah et al. How frequent are vancomycin-resistant enterococci in patients with primary sclerosing cholangitis and ulcerative colitis treated with oral vancomycin?. Indian J Gastroenterol. https://doi.org/10.1007/s12664-022-01286-9 . Notes: Evaluation of incidence of VRE via rectal swab in 7 adult PSC/IBD patients treated with oral vancomycin. 2021 - F Balouch et al "Paediatric Primary Sclerosing Cholangitis-Ulcerative Colitis. Audit of 2 year Outcome with Oral Vancomycin Treatment". Gastroenterology, May;160(6):S-44. Full text available without subscription at doi.org/10.1016/S0016-5085(21)00852-0 . Notes: 29 children with PSC and IBD (most failing conventional medical therapy for UC) treated with oral vancomycin. Study evaluates remission of UC over 2-year follow-up. 2021 - SL Britto et al. “Microbiome Responses to Vancomycin Treatment in a Child With Primary Sclerosing Cholangitis and Ulcerative Colitis.” ACG case reports journal vol. 8,5 e00577. 11 May. Full text available without subscription at doi:10.14309/crj.0000000000000577 . Notes: Case study of 7-year-old PSC/IBD patient with longitudinal microbiome analysis. 2021 - CW Buness et al. “Successful response of primary sclerosing cholangitis and associated ulcerative colitis to oral vancomycin may depend on brand and personalized dose: report in an adolescent.” Clinical journal of gastroenterology vol. 14,2 : 684-689. Full text available without subscription at doi:10.1007/s12328-020-01296-0 . Notes: Case study of adult with PSC/IBD who started oral vancomycin treatment as a teen. 2021 - A Di Giorgio et al. “Use of oral vancomycin in children with autoimmune liver disease: A single centre experience.” World journal of hepatology vol. 13,12:2113 2127 doi:10.4254/wjh.v13.i12.2113 Notes: Retrospective evaluation of response of PSC for 12 pediatric PSC patients taking oral vancomycin after failing standard immunosuppresion at an Italian centre. 2021 - AU Rahman et al. The role of oral vancomycin in inducing remission for biologic-experienced ulcerative colitis with concomitant primary sclerosing cholangitis and liver transplantation. Clin J Gastroenterol 14, 159–164. https://doi.org/10.1007/s12328-020-01272-8 Notes: Case study of an adult post-transplant PSC/IBD patient; reports on UC response to oral vancomycin treatment after failing conventional UC medications. 2019 - A Dao et al. Letter to the Editor: “Oral Vancomycin Induces and Maintains Remission of Ulcerative Colitis in the Subset of Patients With Associated Primary Sclerosing Cholangitis.” Inflammatory bowel diseases vol. 25,7: e90-e91. Letter can be found without subscription at doi:10.1093/ibd/izz027 Notes: Retrospective chart review of response of UC in 8 PSC/UC patients seen at an IBD clinic (at US hospital) who were treated with oral vancomycin. No evaluation of response of PSC to treatment. Follow-up time from 9-36 months. 2019 - LZ Tan et al. “Oral vancomycin induces clinical and mucosal remission of colitis in children with primary sclerosing cholangitis-ulcerative colitis.” Gut vol. 68,8: 1533-1535. doi:10.1136/gutjnl-2018-316599 Notes: Retrospective study of IBD response to oral vancomycin over median 8.1 months on 7 pediatric PSC/IBD patients. 2018 - GP de Chambrun et al. “Oral vancomycin induces sustained deep remission in adult patients with ulcerative colitis and primary sclerosing cholangitis.” European journal of gastroenterology & hepatology vol. 30,10: 1247-1252. doi:10.1097/MEG.0000000000001223 Notes: Case studies of 3 adult PSC/IBD patients taking oral vancomycin. 2017 - P Hey et al. “Efficacy of oral vancomycin in recurrent primary sclerosing cholangitis following liver transplantation.” BMJ case reports vol. 2017 bcr2017221165. 25 Sep., doi:10.1136/bcr-2017-221165 Notes: Adult post-transplant PSC. r-PSC diagnosed 3 years after transplant. 2014 - S Brost et al. "Orale Therapie mit Vancomycin als Therapieoption bei Primär Sklerosierender Cholangitis" Z Gastroenterol 2014; 52 - KG125 DOI: 10.1055/s-0034-1386147 Notes: Adult PSC patient started on oral vancomycin for C-diff infection. Reports on biochemistry and symptoms after starting, stopping, and starting oral vancomycin again. 2013 -DN Abarbanel et al. “Immunomodulatory effect of vancomycin on Treg in pediatric inflammatory bowel disease and primary sclerosing cholangitis.” Journal of clinical immunology vol. 33,2: 397-406. Full text available without subscription at doi:10.1007/s10875-012-9801-1 . Notes: Examination of the in vivo immunological changes that occur during oral vancomycin treatment of the 14 children with PSC and IBD in the case series described in Davies et al 2008 . 2013 - YK Davies et al. “Successful treatment of recurrent primary sclerosing cholangitis after orthotopic liver transplantation with oral vancomycin.” Case reports in transplantation vol.: 314292. Full text available without subscription at doi:10.1155/2013/314292 . Notes: Case study of one pediatric post-transplant PSC patient who started oral vancomycin after developing recurrent PSC. 2008 - YK Davies et al. “Long-term treatment of primary sclerosing cholangitis in children with oral vancomycin: an immunomodulating antibiotic.” Journal of pediatric gastroenterology and nutrition vol. 47,1: 61-7. Full text available without subscription at doi:10.1097/MPG.0b013e31816fee95 Notes: Case studies of 14 pediatric PSC patients treated with oral vancomycin. 1998 - KL Cox and KM Cox. “Oral vancomycin: treatment of primary sclerosing cholangitis in children with inflammatory bowel disease.” Journal of pediatric gastroenterology and nutrition vol. 27,5: 580-3. Full text available without subscription at doi:10.1097/00005176-199811000-00015 . Notes: Case studies of 3 pediatric PSC patients treated with oral vancomycin. Commentary/Letters to the Editor/etc. 2024 - Shah A, Holtmann G. Editorial: Is it time to include oral vancomycin in therapeutic guidelines for primary sclerosing cholangitis-associated inflammatory bowel disease? Aliment Pharmacol Ther. 2024 May; 59(10):1290-1291. doi: 10.1111/apt.17990. PMID: 38652768. https://pubmed.ncbi.nlm.nih.gov/38652768/ 2023 - J Halma et al. The Time is Ripe for a Randomized Controlled Trial of Oral Vancomycin in Primary Sclerosing Cholangitis. Clinical Gastroenterology and Hepatology LETTER TO THE EDITOR| VOLUME 21, ISSUE 10, P2699-2700, SEPTEMBER 2023, https://pubmed.ncbi.nlm.nih.gov/36442726/. Notes: Full text here .

< 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

The VIADUCT Newsletter Join our Email List! The Viaduct - Issue 10, Winter 2026 Dear Registry Community, 2025 has been a banner year for the Patient Registry. Between important new surveys, Registry-led publications, external research support, and the launch of Spanish and French translations, the Registry is reaching ever greater heights. At the heart of the Registry’s success, making everything we do possible, is your active participation in the Registry. With 223 new participants in 2025, the Registry now represents over 2,770 people living with PSC f Feb 10 The Viaduct - Issue 9, Fall 2025 Living with PSC, Leading with Hope You might notice this issue of The Viaduct has a new look, in more ways than one! As your new Registry director, it is my honor to serve you in fulfilling the Registry’s mission to facilitate, expedite, and advance patient-centered research towards treatments and a cure for PSC. When I was diagnosed with PSC, I felt the same as many of you did: hopeless, uncertain, fearful. As I pored over medical websites, papers, and personal stories, two Sep 23, 2025 The VIADUCT - Issue 8 Issue 8 - Spring 2025 In this issue: From the Registry Director • Cholangiocarcinoma (CCA) Survey • WIND-PSC Enrolling Patients • Clinical Trial News • External Studies and Surveys • Symptom Assessment Project • Registry Superstars • Learning from Registry Data From the Registry Director Rachel Gomel (left) and Mary P Vyas (right) Dear Registry Community, Sixteen years have gone by since the PSC Partners Patient Registry was just a lofty idea, a dream I believed could turn in Apr 9, 2025 The VIADUCT Newsletter Issue 7 Issue 7 - Winter 2025 In this issue: From the Registry Director • A New Era • Your Data in Publication - 2024 • Clinical Trial News • Registry Data Presented at TLM • Your Powerful Impact • PSC Partners Registry and Other Registries From the Registry Director Dear Registry Participants, This has been an incredibly fruitful year for the Registry. Between a move to the new and improved Registry platform, several publications making use of Registry data, and the launch of two ne Dec 28, 2024 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 Feedback • Oral Vancomycin & Insurance Coverage • European Clinicians Survey Results • Call to Action: What is the Registry Not Asking? • Highlights: Clinical Trials & Studies From the Registry Director Dear Registry Participants, PSC Partners and the PSC Partners Patient Registry are moving at rapid speed through involvement in large internatio Jun 29, 2023 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 PSC Research • The ChiLDRen Study • Gilead's PRIMUS Trial Terminated • Patient-friendly Clinical Trial Design • Highlights of the 2022 International Liver Congress • Meet PSCP's Ruth-Anne Pai, PhD • Clinical Trials Are Recruiting From the Registry Director Dear Registry Participants, Whether you are a person living with PSC, or the relat Oct 29, 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 Partners WIND Natural History Project • ROADMAP Wrap-up • PSC Research: Collaborative Efforts From the Registry Director Dear Registry Participants, Excitement is mounting as we approach the 18th PSC Partners Annual Conference, which is being planned in collaboration with Brigham and Women's and Massachusetts General hospitals (Harvard Univers May 24, 2022 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 Poster Presentation: AASLD Liver Meeting, Nov. 2021 • Three Clinical Trials Currently Recruiting • Three Minutes with Frank Sasinowski • AASLD Patient Track Recap • Update on the ROADMAP Initiative From the Registry Director Dear Registry Participant, The holidays are here and, once again, COVID-19 is affecting our lives in many different Dec 1, 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 of The VIADUCT to you! Email us at any time with your feedback, input or questions at registrycoordinator@pscpartners.org . In this issue: From the Registry Director: Moving Research Forward Clinical Trials: Stay informed with Registry Resources | Fred Sabernick Shares His First-Hand Experience Boston: An Emerging Hub for PSC Research Home Aug 23, 2021

< Back 2025. AASLD The Liver Meeting Abstract and Poster: 4540: "IT'S MORE LIKE A DULL, ACHY PAIN THAT'S JUST CONSTANT": LIVER PAIN EXPERIENCES IN ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS (PSC) TO INFORM MEASURE DEVELOPMENT Authors: Donna Evon, Lauren Wright, Laura Mkumba, Nicole Lucas, Kaya Merkler, Ricky Safer, Joanne Hatchett, Rachel Gomel, Stephen Rossi, Mark Swain, Sasha Deutsch-Link, Bryce Reeve Abstract: Background: An in-depth understanding of experiences with liver pain associated with chronic liver disease is virtually non-existent, especially for individuals living with PSC. The development of a PSC-specific liver pain measure suitable for clinical trials relies upon understanding key characteristics of liver pain in this population. Methods: Adults with PSC were recruited through the PSC Partners Patient Registry and hepatology experts in the U.S. Participants were phone screened for the presence of multiple PSC-related symptoms. Purposeful sampling was used to recruit a representative sample of 17 adults for in-depth qualitative interviews about key PSC symptoms experienced in the last month, including liver pain. Interviews were conducted virtually, audio-recorded, transcribed, and analyzed using NVivo software. Results: Ten out of 17 participants who reported liver pain as a prominent PSC symptom described their experiences in detail, while another three spontaneously mentioned liver pain during their interviews. Participants were, on average 43 years old (range: 27-65), 50% male, 60% white, 30% black, 10% Asian and 30% had inflammatory bowel disease (IBD). Participants’ experiences of their liver pain are found in the Table below. Participants described the location of PSC-associated liver pain generally in the right upper quadrant area, but said it can extend around the side and back. Eleven of 13 described liver pain as “stabbing” or “sharp.” Eight described their pain as “achy” or “dull.” The sharp/stabbing pain is typically brief, while the achy/dull pain is more persistent. All but one participant used the word “pain” to describe their sensations. Liver pain can “come and go” intermittently, happens at random, and seems unassociated with specific triggers. Some reported pain daily, others weekly, and most reported pain occurring outside of cholangitis attacks. Participants were able to differentiate PSC-related liver pain versus abdominal pain from other causes (including IBD) based on location and type of pain, timing, response to medications, and laboratory/clinical markers. Liver pain interfered with sleep and caused anxiety, but relative to fatigue and brain fog, it appears to interfere less with daily functioning. Participants tended to “ push through it.” Conclusion: Dull/achy and sharp/stabbing liver pain may be more frequent and severe in symptomatic adults with PSC than previously recognized. These in-depth qualitative findings will inform the development of a de novo PSC-specific liver pain measure, inclusive of heterogeneity of experiences, and suitable for future PSC research studies. Previous Next

Learn more about the Registry's policies and procedures for researchers to obtain the approved types of de-identified registry data and/or confidentially send recruitment trial requests to eligible Registry participants. Data Access Policy Overview Introduction PSC Partners Seeking a Cure (PSC Partners) is committed to providing investigators and industry an opportunity to access the PSC Partners Patient Registry (Registry) data, and to assist in study recruitment while maintaining Registry participant privacy. Qualified non-profit investigators will be accorded Registry data without charge, while industry will be required to pay PSC Partners a license fee for the data to assist in covering some of the costs of maintaining the registry. In making data available to investigators, PSC Partners is bound by its responsibility to guard the confidentiality of registry participants. This policy provides information and guidelines for investigators and industry sponsors wishing to request access to de-identified patient data residing in the Registry. This policy requires that Registry data use remain ethical, purposeful and consistent with each original study proposal and request to access data. Levels of Data Four (4) levels of data can be requested by investigators and approved by the Registry Committee: Graphs of de-identified aggregate data. This data can be requested from the Registry Committee by emailing registryresearch@pscpartners.org . Case-by-case de-identified data . This level of data requires a project proposal, IRB approval or exemption and the approval of the Registry Director and the Registry Committee Chair. To be delivered on a password-protected spreadsheet or .csv file. Request for additional patient data through emailed questionnaire. This level of data requires a project proposal, IRB approval or exemption and the approval of the Registry Committee. [This is when the shared Registry data is insufficient and the investigator requests the implementation of their own survey.] Recruitment of registrants for clinical trials. Request to access individual subjects requires a project proposal, IRB approval and the approval of the Registry Committee. The Registry curator can perform queries based on the inclusion/exclusion criteria of a study and send potential subjects information about a study for which they may qualify. The Registry may provide the subject/caregiver a recruitment flier, informed consent form, and/or contact information for the site(s) conducting the study. Personal identifiers or subject contact information will not be provided to investigators. It is the responsibility of the consenting registry participants to contact the investigators. Requestor's Responsibilities Regarding Data Use The specific purpose for the data use must be agreed to in writing by the investigator and his/her employer institution (“Institution”) or the industry sponsor requesting the material and the selected representatives of the Registry Committee including medical and patient representatives (Registry Committee) for the approval of the request. The investigator and Investigator’s Institution or industry sponsor (if any) must sign this Data Use Agreement, agreeing to abide by the Researcher Data Access Policy. The investigator must provide an electronic copy of an IRB approval, or for surveys, IRB exemption (see Levels of Data below), a study proposal including the specific aims of the study/clinical trial, and the name and qualifications of the research center. The rationale for selection of the population inclusion and exclusion criteria must be described in the project proposal. Other information may be required as requested by the Registry Director. The investigator and investigator’s Institution or industry sponsor (if any) must agree in writing that the data will be used only for the stated purpose and only by the requesting party and may not be passed on or shared with a third party. (a) This Data Use Agreement is not transferable. (b) Data will not be used to establish the individual identities of any of the subjects from whom data is obtained. (c) Investigator, team, Institution and industry sponsors, if applicable, employees, and other research staff members must agree not to transfer data to any third party entity or any individual. The investigator, Institution and industry sponsor (if any) must agree that they will not sell or distribute the data and will only share the data with research staff members. The full team of individuals involved in the study are subject to the terms of the Data Use Agreement. The data may be used only for the time period specified in the approved request. Approval for access to the Registry data will be for an interval of no more than one (1) year from the date of approval, and Investigators must re-apply and submit a progress report to be approved by the Registry Committee in order to continue to use Registry data to recruit, or to continue to use the data for an approved study. The impact of research on people living with PSC is to be a key consideration for each project. Best ethical practices for ensuring the interest of the patient are to be applied. Any publication, oral and written presentations, or disclosures resulting from any and all analyses of the Registry data or based on the Registry data shall be subject to prior review by the Registry Director, must specify PSC Partners as the source of data, and are restricted to the scope of the approved study proposal. A copy of the final publication(s) must be sent to the Registry Director within 30 days of publication. Investigators, their Institution and industry sponsors accessing and using patient data must agree in writing to assume full responsibility for any and all uses of such data. Recipients of Registry data agree not to use it in advertising, publicity, or for purposes other than the purposes for which access to Registry data was granted, and not to use the logo, symbol, or other image of PSC Partners without its prior written consent. Steps to Request Access to Data An investigator and industry sponsor will be permitted access to Registry data only after meeting the following requirements: Contact the Registry via email at registryresearch@pscpartners.org . The Registry Team will provide a Data Access Form and Data Use Agreement. Complete the Data Access Form confirming that the investigator and investigator’s Institution or industry sponsor and research staff will abide by the Data Access Policy. Provide a project proposal, including: (a) Study proposal including the specific aims of the study (b) Name and qualifications of the investigator(s); include the full research and team of all individuals involved in this study with their qualifications. (c) Research center (d) In the case of a clinical trial: (i) Description of the procedures that will protect the confidentiality and security of Registry participants (ii) Sample study recruitment letter (iii) Consent form (iv) Rationale for selection of the population inclusion and exclusion criteria should be described in the project proposal (e) Disclosure of financial conflict of interest (f) Description of the data being requested Provide IRB approval with the project proposal if the requested data involves de-identified case-by-case results, a request for additional de-identified data through an e-mailed questionnaire, and/or a request for participants for a clinical trial. Receive written approval of the Registry Committee if the proposal requests research access to the Registry data. Submit a declaration of interest (included in the Data Access Form and Data Use Agreement). This may be submitted following a positive review of the project proposal if so desired. In the case of surveys to all or selected Registry participants, coordinate with the Registry staff on both the timing and content of the email to be sent to Registry participants informing them of the opportunity to participate in the Registry Committee-approved survey. If you have any questions r egarding these policies or regarding t he procedure to request access to Registry data, please contact us at registryresearch@pscpartners.org .