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Posts (15)

Mon, Jul 29 8:41am · IBD: A Comorbidity of PSC in Primary Sclerosing Cholangitis (PSC)

 

2019-08-13 Comorbidity PSC

The majority of patients with the PSC have Inflammatory Bowel Disease (IBD). In this presentation, Dr. Laura Raffals, Associate Professor of Medicine, Mayo Clinic College of Medicine provides a comprehensive review of the epidemiology, diagnosis, outcomes and management of IBD among PSC patients.

View the powerpoint presentation from Dr. Raffals

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Mon, Jul 15 10:37am · PSC and Inflammatory Bowel Disease (IBD): Women’s Health Issues in Primary Sclerosing Cholangitis (PSC)

PSCandIBDandWomensHealth

Female patients with the PSC and inflammatory bowel disease (IBD) have unique health and disease issues to deal with.

During the 15th Annual Conference of the “PSC Partners Seeking a Cure”“, Dr. Alina Allen, Assistant Professor of Medicine, Mayo Clinic College of Medicine, and Dr. Laura Raffals, Associate Professor of Medicine, Mayo Clinic College of Medicine, provided an excellent overview of the specific situations (i.e., conception, pregnancy, breastfeeding, etc) that are relevant to women with PSC and IBD and offer management approaches and advice.

View the powerpoint presentation from Dr. Allen and Dr. Raffals.

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Mon, Jul 1 4:13pm · Genes and Environment in PSC in Primary Sclerosing Cholangitis (PSC)

Gene and Environment Pic

On May 21-23, 2019, the 15th Annual Conference of the “PSC Partners Seeking a Cure” took place in Rochester, Minnesota. More than 315 patients and family members attended the meeting. We are honored and grateful that “PSC Partners Seeking a Cure” choose Rochester and Mayo Clinic as their destination for 2019. We are inspired by their mission and efforts. Starting this week and in the upcoming months we will include in our newsfeed section selected presentations of this year’s conference delivered by faculty of Mayo Clinic. A link to the first presentation’s slides, entitled: Genes and Environment in PSC, is provided below.

View the powerpoint presentation from Dr. Lazaridis

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Mon, Jun 3 9:53am · PSC literature elsewhere: Ursodeoxycholic acid therapy in pediatric PSC in Primary Sclerosing Cholangitis (PSC)

2019-05-31 Patient taking medicine

Recently, we have made the decision to include periodically in our PSC blog commentaries of selected clinical studies published by other groups. This tactic aims at keeping the followers of this blog informed about current efforts of the academic community at large to better understand and treat PSC.

In a retrospective study published in the Journal of Pediatrics in June 2019 (see below), Mark Deneau et al., examined the clinical factors predicting normalization of gamma glutamyltransferase (GGT) in children affected by PSC following therapy with ursodeoxycholic acid (UDCA).

In this large study, 344 PSC patients from 46 medical centers were identified with a serum GGT more than 50IU/L at the time of diagnosis who had also repeat testing one year later. Subsequently, 81 patients were excluded because they were not taking UDCA or developed complications of the disease 3 months following the diagnosis. The remaining 263 patients (60% were male with a median age of 12.1 years) were followed for a median of 5.4 years. The median dose of UDCA was 15mg/kg daily. 122 of 263 patients (46%) had normalization of GGT at 1 year. At the time of diagnosis, the group of patients that had favorable biochemical response on UDCA had a lower prevalence of Crohn’s disease, lower total bilirubin, lower aspartate aminotransferase to platelet ratio index, and greater serum albumin level. The presence of small or large duct PSC, or the concurrence of autoimmune hepatitis was not associated with normalization of GGT. The 5-year survival of patients with normalization of UDCA while on UDCA was 99% compared to 77% of patients who did not achieve normalization.

This was a large study from a diverse number of medical centers and the authors should be congratulated for this significant effort. It appears that UDCA normalizes GGT in a number of pediatric patients and these have identifiable clinically relevant factors. The study also suggests that lack of GGT normalization, while on UDCA, should make providers to discontinue therapy although compliance with this drug was unknown. The limitations of the study include its retrospective nature, the fact that it did not account for severity and duration of disease, as well as concurrent immunosuppressive therapy of the patients. In summary, this is an interesting study but we still need better objective biomarkers of PSC severity in order to improve our knowledge on treatment response in PSC patients.

The pdf of the published article can be found here.

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Mon, May 20 11:25am · Efficacy and safety of curcumin in PSC: an open label pilot study in Primary Sclerosing Cholangitis (PSC)

2019-06-04 Curcumin

In this month’s issue of the Scandinavian Journal of Gastroenterology John E. Eaton et al., reports on the results of a 12-week open-label pilot study to examine whether curcumin, a naturally occurring compound, could reduce the markers of cholestasis in patients with PSC. Curcumin is the principal curcuminoid of the rhizome turmeric (Curcuma longa) and is believed to have anti-inflammatory and anti-oxidant effects. Curcumin may function as a free radical scavenger and reduce oxidative damage through several proposed mechanisms. In two separate rodent models of cholestasis curcumin was associated with a reduction in liver enzymes and improvement in hepatic histology.

In this pilot study by John E. Eaton et al., patients with PSC and a serum alkaline phosphatase (SAP) greater than 1.5 x the upper limit of normal (ULN) received curcumin 750 mg orally twice daily for 12 weeks. The primary study endpoint was the proportion of subjects who had a reduction of SAP to less than 1.5 x ULN or a 40% reduction in SAP between baseline and week 12. Secondary study endpoints included changes in serum aspartate aminotransferase, total bilirubin, Mayo PSC risk score and self-reported health questionnaires. The authors screened 258 patients with PSC in order to enroll 15 subjects. The most common reason for subject exclusion was SAP < 1.5 x ULN (n = 98 or 38%). Curcumin did not result in a significant median (interquartile range) change in SAP x ULN [3.43 (2.10–4.32) to 2.46 (1.89–4.41), p = .36] and only 20% (3/15) of subjects achieved the primary study endpoint. There was no significant change in the secondary study endpoints. The authors did not report any serious adverse events among the enrolled patients. In summary, curcumin was well tolerated by the study participants but it was not associated with significant improvement of cholestasis or symptoms.

In this first clinical trial of curcumin among patients with PSC only 20% of the subjects, rather the target of 30%, met the primary study endpoint. Moreover, the study emphasizes that a SAP of <1.5 x ULN is common among patients with PSC. This observation, which highlights the known fluctuation of SAP during the course of PSC, makes this biomarker suboptimal as a tool to predict the progression of disease. Thus, there is an urgent need to discover more reliable biomarkers of PSC in order to better understand the disease progression and ultimately to improve its therapy.

The PDF of the published article can be found here.

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Mon, May 6 12:55pm · Cholangiocyte Pathobiology in Primary Sclerosing Cholangitis (PSC)

2019-05-31 Figure 4

 

In a review published in Nature Reviews Gastroenterology and Hepatology this month (see below), Jesus Banales et al., provides an excellent and comprehensive overview of cholangiocyte pathobiology as relates to the development of biliary diseases like PSC. Initially, the authors emphasize on the normal function of cholangiocytes, the cells that line the bile ducts inside and outside the liver. Subsequently, the review focuses on the role of cholangiocytes in biliary development, inflammation, immunology and fibrosis.

It is accepted that cholangiocytes are critical in hepatic health and disease although they represent only about 5% of the liver cells. They are the target cells in PSC and other cholangiopathies. Over the past two decades, we have witnessed a significant growth of the knowledge on cholangiocyte biology as this information is well captured in this review. Yet, elucidating further the ecosystem of the cholangiocytes will help us to improve our understanding of PSC.

The PDF of the published article can be found here.

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Mon, Mar 25 10:52am · 15th annual conference for PSC patients and caregivers in Rochester, MN in Primary Sclerosing Cholangitis (PSC)

2019-03-25 Mayo2

We are privileged to host in Rochester, Minnesota the 15th annual conference for PSC patients and their caregivers (June 20-23, 2019). In a partnership with the PSC Partners Seeking a Cure we have created an exciting and informative program (please see the attached agenda). During this conference, we will highlight our current state of-the-art research efforts, and existing and future clinical trials for PSC, as well as practical issues about the management of, and dealing with PSC.

The PDF of the 2019 PSC Partners Conference Agenda can be found here.

 

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Mon, Feb 25 5:10pm · Rare Disease Day February 28th, 2019 in Primary Sclerosing Cholangitis (PSC)

2018-02-24 Rare Disease Day

This Thursday, February 28th, we celebrate: “Rare Disease Day”. Therefore, we dedicate this week’s post to the anniversary of Rare Disease Day in an effort to raise awareness about rare diseases.

Rare diseases are individually rare but collectively common. In the United States, a “rare disease” is defined as one that affects fewer than 200,000 people. Today, there are as many as 7,000 rare diseases and new ones are discovered every year. About 1 out of 11 Americans are living with a rare disease. In fact, the total number of Americans living with a rare disease is estimated between 25-30 million. Approximately 80% of rare diseases are not acquired, they are inherited and are caused by mutations or defects in genes. In addition, over 50% of the rare diseases remain sub-clinical in childhood and become clinically apparent and/or diagnosed during adulthood.

Rare diseases are often referred to as “orphan disease”. Rare or orphan diseases are frequently not pursued by the pharmaceutical industry because they provide little financial incentive for the private sector to make and market new medications. Of interest, some of the most important drug discoveries ever made were founded on the basis of elucidating the genetic cause of a rare disease. For example, statins were developed as the result of the molecular discoveries causing homozygous hypercholesterolemia, which affects about 1 person in a million individuals. Given the lack of financial incentives to treat rare diseases the US Orphan Drug Act in 1983 has been a successful story of health-related legislation in the United States. Through a system of tax credits, government grants, and assistance for clinical research, the Orphan Drug Act has resulted in hundreds of approved orphan medicines treating over millions of patients.

Patients affected by a rare disease have frequently a diagnosis but many rare diseases have no treatment. Extremely rare diseases that lack a diagnosis are termed undiagnosed diseases or diagnostic odysseys. Thus, although virtually all undiagnosed diseases are rare diseases many rare diseases are not undiagnosed diseases.

Primary Sclerosing Cholangitis (PSC) is a rare disease. In the United States, large epidemiological studies on the incidence (i.e., the rate of occurrence of new cases of a disease per year) and prevalence (i.e., the proportion of all cases of a disease in a population) of PSC are limited. It is estimated that there are about 30,000 to 50,000 patients diagnosed with PSC in this country. However, the actual number of patients living with PSC in the United States is likely higher since individuals that remain undiagnosed or mis-diagnosed are not included in the estimation above.

For all of us who study patients with PSC this is an exciting time for research. We are making progress to discover the cause(s) of the disease and plan to use this knowledge to slow its progression or find a cure. And in this effort, we are not alone. There is a dedicated patient support group: the “PSC Partners Seeking a Cure”, which provides education and support to patients with PSC, family members and caregivers as well as promotes more research for the disease. Moreover, there is an unwavering support from benefactors and the NIH to further study PSC with the aim to discover a cure for this elusive disease. These powers are coming together offering hope and optimism for patients with PSC. There is a robust momentum in place and this is needed for a major breakthrough that will transform in a positive way the care of patients with PSC.

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