Stratification of hepatocellular carcinoma risk in primary biliary cirrhosis: a multicentre international study

Trivedi, Palak; Lammers, Wim J. E. P.; Buuren, Henk R. van; Parés, Albert; Floreani, Annarosa; Janssen, Harry L.A.; Invernizzi, Pietro; Battezzati, Pier Maria; Ponsioen, Cyriel Y.; Corpechot, Christophe; Poupon, Raoul; Mayo, Marlyn J.; Burroughs, Andrew K.; Nevens, Frederik; Mason, Andrew L.; Kowdley, Kris V.; Lleo, Ana; Caballería, Llorenç; Lindor, Keith D.; Hansen, Bettina E.; Hirschfield, Gideon M.

doi:10.1136/gutjnl-2014-308351

Cited by 150 publications

(119 citation statements)

References 39 publications

Supporting

Mentioning

107

Contrasting

Unclassified

Order By: Relevance

“…These data have been confirmed, indicating that 12-month biochemical non-response is associated with increased future risk of developing hepatocarcinoma [30] .…”

Section: Udca As the Specific Therapysupporting

confidence: 73%

Therapy of Primary Biliary Cirrhosis: Novel Approaches for Patients with Suboptimal Response to Ursodeoxycholic Acid

Parés¹

2015

Dig Dis

Self Cite

View full text Add to dashboard Cite

Primary biliary cirrhosis (PBC) is a chronic cholestatic disease of presumed autoimmune pathogenesis, characterized by the inflammation and damage of the intrahepatic intermediate and small bile ducts, which eventually results in cirrhosis. A number of randomized and observational and pilot studies using several agents were carried out in the 80s, but no clear results or even harmful effects were reported. Over the past 2 decades, increasing evidence indicates that ursodeoxycholic acid (UDCA) - 13 to 16 mg/kg/day - is the treatment of choice for patients with PBC. Biochemical response to UDCA, assessed at 1 year, clearly predicts the long-term outcome, since in UDCA, responders survival is similar to that estimated for the matched control population. However, about 40% of patients have incomplete biochemical response and increased risk of progression and decreased survival free of transplantation. Patients with suboptimal biochemical response to UDCA outline the group in whom further single or combined treatments with UDCA are needed. Accordingly, data on the effect of fibrates alone or in combination with UDCA, and budesonide in combination with UDCA have been reported. The combined treatment of UDCA and fibrates in patients without optimal biochemical response to UDCA improves the degree of cholestasis and may minimize the long-term management of these patients. The results of the combined therapy of UDCA with budesonide are appealing but they should be established in large randomized trials. The effect of new agents such obeticholic acid are promising, since the addition of this farnesoide-X-receptor agonist bile acid in patients with stable UDCA dosage and increased alkaline phosphatase levels results in an improvement of cholestasis as compared to placebo, with a parallel decrease of aminotransferases and immunoglobulin M, as well as one surrogate marker of bile acid synthesis. New molecular therapies are currently being investigated.

show abstract

“…These data have been confirmed, indicating that 12-month biochemical non-response is associated with increased future risk of developing hepatocarcinoma [30] .…”

Section: Udca As the Specific Therapysupporting

confidence: 73%

Therapy of Primary Biliary Cirrhosis: Novel Approaches for Patients with Suboptimal Response to Ursodeoxycholic Acid

Parés¹

2015

Dig Dis

Self Cite

View full text Add to dashboard Cite

show abstract

“…18 These data were used to develop a validated scoring system to predict transplant-free survival in ursodeoxycholic acid-treated PBC patients and to elucidate predictors for development of HCC. 19,20 International successes like these emphasize that combining several national databases constitutes a unique opportunity to obtain the power to execute studies.…”

Section: Designmentioning

confidence: 99%

Creating an effective clinical registry for rare diseases

D’Agnolo

Kievit²,

Andrade

et al. 2015

United European Gastroenterology Journal

View full text Add to dashboard Cite

The exposure of clinicians to patients with rare gastrointestinal diseases is limited. This hurts clinical studies, which impedes accumulation of scientific knowledge on the natural disease course, treatment outcomes and prognosis in these patients. An excellent method to detect patterns on an aggregate level that would not be possible to discover in individual cases, is a registry study. This paper aims to describe a template to create a successful international registry for rare diseases. We focus mainly on rare hepatic diseases, but lessons from this paper serve other fields in medicine, as well.

show abstract

“…A recent study conducted by [44]. The role of male sex in the development of HCC in PBC has also been found in a representative cohort of Chinese and Japanese patients with a long follow-up [45,46].…”

Section: Causes Of Deathmentioning

confidence: 99%

Geoepidemiology and changing mortality in primary biliary cholangitis

et al. 2017

Self Cite

View full text Add to dashboard Cite

show abstract

Stratification of hepatocellular carcinoma risk in primary biliary cirrhosis: a multicentre international study

Abstract: This uniquely powered, internationally representative cohort robustly demonstrates that 12-month biochemical non-response is associated with increased future risk of developing HCC in PBC. Such risk stratification is relevant to patient care and development of new therapies.

Cited by 150 publications

References 39 publications

Therapy of Primary Biliary Cirrhosis: Novel Approaches for Patients with Suboptimal Response to Ursodeoxycholic Acid

Therapy of Primary Biliary Cirrhosis: Novel Approaches for Patients with Suboptimal Response to Ursodeoxycholic Acid

Creating an effective clinical registry for rare diseases

Geoepidemiology and changing mortality in primary biliary cholangitis

Contact Info

Product

Resources

About