Registry Impact

 

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Registered Participants

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Survey Question Responses

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Healthcare Provider Listings

 

The following publications resulted from access to your de-identified PSC Partners Registry data. 

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.

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."

AASLD Poster 2019

Data from PSC Partners Patient Registry

Abstract can be found here

Authors: Buness JG, Ali AH, Tabibian JH, Buness CW, Cox KL, Lindor KD

Journal: [published online ahead of print, 2019 Aug 30]. Am J Ther. 2019;10.1097/MJT.0000000000001065. doi:10.1097/MJT.0000000000001065

Excerpts:

"Primary sclerosing cholangitis (PSC) is linked to inflammatory bowel diseases (IBD). Evidence suggests an association between the gut microbiome and PSC. However, the putative relationship between exposure to antibiotics and onset of PSC has never been reported. We observed 3 cases in which patients without antecedent liver or bowel issues developed symptoms leading to diagnosis of IBD and subsequently PSC after being exposed to doxycycline. We aimed to identify, through the PSC Partners national patient registry, additional cases of PSC in which there is a temporal relationship between exposure to doxycycline and onset of PSC or PSC-IBD."

Pubmed.gov listing and abstract

Authors: Zobair M. Younossi Arian Afendy Maria Stepanova Andrei Racila Fatema Nader Rachel Gomel Ricky Safer William R. Lenderking Anne Skalicky Leah Kleinman Robert P. Myers G. Mani Subramanian John G. McHutchison Cynthia Levy Christopher L. Bowlus Kris Kowdley Andrew J. Muir

Journal: AASLD Hepatology First published:20 November 2017 https://doi.org/10.1002/hep.29664 https://doi.org/10.1002/hep.29664

Excerpts:

"Our objective was to develop and validate a PSC‐specific patient‐reported outcome (PRO) instrument. We developed a 42‐item PSC PRO instrument that contains two modules (Symptoms and Impact of Symptoms) and conducted an external validation."

"Conclusion:  PSC PRO is a self‐administered disease‐specific instrument developed according to U.S. Food and Drug Administration guidelines. This preliminary validation study suggests good psychometric properties. Further validation of the instrument in a larger and more diverse sample of PSC patients is needed."

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."

EASL Poster 2016

Data from PSC Partners Patient Registry

 

AASLD Liver Meeting Abstract 1112

Authors: Gomel, Rachel & Safer, Ricky & Lindor, Keith & Everson, Gregory & Bowlus, Christopher

Journal: Gastroenterology. 150. S1058-S1059. 10.1016/S0016-5085(16)33574-0 https://doi.org/10.1016/S0016-5085(16)33574-0

Excerpts:

"Conclusions: 

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"

AASLD Poster 2015

Data from PSC Partners Patient Registry

The Patient Registry enables advocacy on behalf of PSC patients.

Clinical Trial and Study Protocol:

PSC Partners is collaborating with pharmaceutical companies planning and conducting clinical trials by adding your patient perspective to the trial protocol. We also collaborate with researchers on their academic study protocols.

Communication of Trial and Study Opportunities with Patients:

Through the Patient Registry, we are able to share clinical-trial and study information with the PSC community. Those participants who have elected to receive notification from the Registry will be contacted by the Registry if they may be eligible for a trial or study.

Special Projects:

Through the Patient Registry, PSC Partners is able to conduct special projects of importance to the patient community.

In March of 2020, various registries were created to compile data on the effect of COVID-19 on individuals with liver disease. However, none were PSC-specific. PSC Partners believes it will be important to collect information on those of us who have/had/suspect having COVID-19 and hence have launched a patient-reported and IRB-approved COVID-19 PSC survey.

We hope very few of us will have COVID-19, but knowing the impact of COVID-19 on our PSC community may bring a new light on PSC. We will share the aggregated information publicly and only in a de-identified format.

Please participate if you have/had/suspect having COVID-19 and you have PSC or had a liver transplant due to PSC. We invite PSC patient groups worldwide to join us in sharing the survey.

Complete survey here.

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.

The relationship between nutrition and PSC is complex and little is known about it. Diet may have an impact on PSC and/or Ulcerative Colitis (UC), but this has not been well established. Researchers led by by PI investigator Joshua Korzenik, MD at Brigham and Women’s Hospital would like to learn more about the relationship between diet, PSC and UC. Registry participants will be notified if they are eligible for this study which consists of two online surveys.

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.

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 the Emerging Trends Conference in Washington D.C. March 6-7, 2018.


Emerging Trends 2018 Table
Emerging Trends Ricky Presenting

PSC Partners Seeking a Cure Surpasses 1,000 Patients in International Registry and Discovers Unexpected Misdiagnoses and PSC Partners Seeking a Cure and PSC Support Announce a Call to Action for Medical Stakeholders to Better Understand and Diagnose Rare Liver Diseases after 

PBC PSC PR Quote

An unexpected result of enrolling more than 1,000 patients in the registry is that some of those who tried to enroll learned -- after answering a series of questions about their diagnosis and symptoms -- that they do not actually have PSC. Instead, they have primary biliary cholangitis (PBC), a distinctly different disease that also affects the bile ducts.

This misdiagnosis is troubling, given that effective treatment exists for PBC and patients who thought they had PSC may have been missing out on treatment that would slow the progression of PBC.

PSC Partners Seeking a Cure and PSC Support, a nonprofit PSC patient organization based in the UK, have been working on an educational campaign to promote a better understanding and more accurate diagnosis of these two rare liver diseases. Given the similarity of their names, PSC and PBC have been confused in medical publications, among regulatory bodies, and in medical practice -- as well as in individual patient diagnoses, as seen in the PSC patient registry.

With the recent renaming of another liver disease, primary biliary cholangitis (PBC), formerly called primary biliary cirrhosis, an opportunity has arisen to alert clinicians of the potential for confusion and stress the importance of correctly identifying these two distinctly different bile duct diseases, PBC and PSC. Patients with PSC also have a devastating and distinctly increased risk of colon and bile duct cancers, while patients with PBC fortunately do not have increased risk for either of these cancers. Consequently, patients with PSC need special cancer surveillance, as well as advice on how to live with cancer risk in a way that is supportive and not life-limiting.

Excerpts above are from press releases by PSC Partners Seeking a Cure. See full text of related press releases: 2017-04 Press Release and 2016-11 Press Release

 PBCPSCfront  PSCPBCquote2  PBCPSCback

 

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.
Ricky  K Pearlman

 

History of Collaboration:

The PSC Partners Patient Registry has worked, or is working with, to provide the patient voice and support recruitment support. 

    • Mirum Pharmaceuticals
    • HighTide Therapeutics
    • Gilead Sciences
    • CymbaBay Therapeutics
    • NGM Biopharmaceuticals
    • Takeda Pharmaceuticals
    • Intercept Pharmaceuticals
    • Lumena Pharmaceuticals
    • Shire Pharmaceuticals
    • Durect Pharmaceuticals
    • Tobira Therapeutics
    • Conatus Pharmaceuticals
    • GlaxoSmithKline (GSK)
    • Patara Pharma
    • Albireo Pharma