Improved Prognosis of Patients With Primary Biliary Cirrhosis That Have a Biochemical Response to Ursodeoxycholic Acid

Kuiper, Edith M. M.; Hansen, Bettina E.; Vries, Richard A. de; Ouden–Muller, Jannie W. den; Ditzhuijsen, Theo J.M. van; Haagsma, E. B.; Houben, Martin H.; Witteman, Ben J.M.; Erpecum, Karel J. van; Buuren, Henk R. van

doi:10.1053/j.gastro.2009.01.003

Cited by 409 publications

(359 citation statements)

References 18 publications

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“…Several studies evaluating longterm survival of patients have been published and uniformly they indicate that those who demonstrated biochemical response to adequate doses of UDCA for prolonged periods of time have longer survival free of liver transplantation and longer overall survival. [15][16][17][18] Currently, treatment with UDCA in a dose of 13-15 mg/kg/day is recommended as therapy for PBC by the AASLD 19 and is approved for this indication by the U.S. Food and Drug Administration (FDA).…”

Section: Introductionmentioning

confidence: 99%

American Association for the Study of Liver Diseases Endpoints Conference: Design and Endpoints for Clinical Trials in Primary Biliary Cirrhosis

et al. 2010

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PreambleA group of multidisciplinary experts on primary biliary cirrhosis (PBC) and its complications convened on May 31, 2009, under the aegis of the American Association for the Study of Liver Diseases (AASLD) in order to identify the most appropriate design and endpoints for clinical trials of PBC based on current evidence and expert experience. The natural history of PBC was reviewed as well as current therapies. The current approaches to evaluating therapies for disease progression and symptoms as priorities were discussed. Appropriate aspects of trial design including entry criteria, study duration, and appropriate handling of issues such as stratification of subjects and use of ursodeoxycholic acid (UDCA), were identified and discussed. After a full day of presentations, in a consensus manner, appropriate endpoints for clinical efficacy trials regarding PBC and its complications were agreed upon and are reported in this summary.

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Section: Introductionmentioning

confidence: 99%

American Association for the Study of Liver Diseases Endpoints Conference: Design and Endpoints for Clinical Trials in Primary Biliary Cirrhosis

et al. 2010

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“…They estimate the risk of LT or death (overall death for the GLOBE score and liver‐related death for the UK‐PBC risk score) in patients with PBC at specific time points. Both scores outperformed previous response criteria7, 8, 9, 10, 11, 13, 14 in terms of prognostic utility and could potentially help physicians identify patients at high risk of disease progression and in need of second‐line therapy. They have also been validated in patients not treated with UDCA, strongly suggesting that such scoring systems reflect disease activity and stage expressed by the laboratory investigations, regardless of treatment.…”

mentioning

confidence: 93%

“…These patients experience progressive liver disease that may eventually lead to liver failure or hepatocellular carcinoma 6. It is well established that the liver biochemistry on treatment with UDCA strongly predicts long‐term outcomes in PBC 7, 8, 9, 10, 11. The response to treatment with UDCA (so‐called UDCA response) may therefore be defined in terms of the liver biochemistry measured at a specific time (usually 12 months) after starting treatment.…”

mentioning

confidence: 99%

“…The response to treatment with UDCA (so‐called UDCA response) may therefore be defined in terms of the liver biochemistry measured at a specific time (usually 12 months) after starting treatment. Several definitions of the UDCA response have been proposed 7, 8, 9, 10, 11. One of these definitions was the Toronto criteria, and a composite of Toronto along with other criteria were used to define treatment response criteria for POISE, a phase 3 trial of treatment with obeticholic acid (OCA) reported by Nevens et al12 By these criteria, response was defined as alkaline phosphatase (ALP) <1.67 × the upper limit of normal (ULN), ≥15% reduction in ALP, and total bilirubin ≤ULN.…”

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confidence: 99%

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Clinical application of the GLOBE and United Kingdom‐primary biliary cholangitis risk scores in a trial cohort of patients with primary biliary cholangitis

Carbone

Harms

Lammers

et al. 2018

Hepatology Communications

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The GLOBAL Primary Biliary Cholangitis (PBC) Study Group and United Kingdom‐PBC (UK‐PBC) Consortium have demonstrated that dichotomous response criteria are not as accurate as continuous equations at predicting mortality or liver transplantation in PBC. The aim of this analysis was to assess the clinical utility of the GLOBE and UK‐PBC risk scores using data from POISE, a phase 3 trial investigating obeticholic acid (OCA) in patients with PBC. Data (N = 216) at baseline and month 12 were used to calculate the GLOBE and UK‐PBC risk scores to assess the projected change in risk with OCA versus placebo. Additionally, the benefit of OCA was assessed in patients not meeting the POISE primary endpoint. Both the GLOBE and UK‐PBC risk scores predicted a significant reduction in long‐term risk of death and liver transplantation after OCA treatment (P < 0.0001). The differences in the relative risk reduction from baseline in the 10‐year event risk after 1 year for OCA 10 mg versus placebo was 26% (GLOBE) and 37% (UK‐PBC). The scores also predicted a significantly decreased risk in patients treated with OCA who did not meet POISE response criteria after 1 year of treatment compared to an increased risk with placebo (P < 0.0001). Conclusion: This analysis demonstrates the use of the GLOBE and UK‐PBC risk scores to assess risk reduction of a cohort treated with OCA. While validation of this risk reduction in studies with clinical outcomes is needed, this study highlights the potential use of these scores in individualizing risk prediction in PBC both in clinical practice and therapeutic trials. (Hepatology Communications 2018;2:683‐692)

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“…However, given the known protracted clinical course of early stage PBC, a much longer follow-up would be necessary to demonstrate survival benefit in this population. Since then, numerous observational studies from Europe and North America have shown that treatment with UDCA is associated with excellent survival free of LT, especially patients with early histological stage and who have biochemical response to UDCA (143,(148)(149)(150)(151) . The decline in number of patients receiving liver transplant for PBC in Europe and North America is seen as further evidence of the survival benefit provided by long-term use of UDCA in PBC (152,153) .…”

Section: Treatment With Ursodeoxycholic Acidmentioning

confidence: 99%

Brazilian society of hepatology recommendations for the diagnosis and management of autoimmune diseases of the liver

Bittencourt

Cançado

Couto

et al. 2015

Arq. Gastroenterol.

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-In order to draw evidence-based recommendations concerning the management of autoimmune diseases of the liver, the Brazilian Society of Hepatology has sponsored a single-topic meeting in October 18th, 2014 at São Paulo. An organizing committee comprised of seven investigators was previously elected by the Governing Board to organize the scientific agenda as well as to select twenty panelists to make a systematic review of the literature and to present topics related to the diagnosis and treatment of autoimmune hepatitis, primary sclerosing cholangitis, primary biliary cirrhosis and their overlap syndromes. After the meeting, all panelists gathered together for the discussion of the topics and the elaboration of those recommendations. The text was subsequently submitted for suggestions and approval of all members of the Brazilian Society of Hepatology through its homepage. The present paper is the final version of the reviewed manuscript organized in topics, followed by the recommendations of the Brazilian Society of Hepatology.

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Improved Prognosis of Patients With Primary Biliary Cirrhosis That Have a Biochemical Response to Ursodeoxycholic Acid

Cited by 409 publications

References 18 publications

American Association for the Study of Liver Diseases Endpoints Conference: Design and Endpoints for Clinical Trials in Primary Biliary Cirrhosis

American Association for the Study of Liver Diseases Endpoints Conference: Design and Endpoints for Clinical Trials in Primary Biliary Cirrhosis

Clinical application of the GLOBE and United Kingdom‐primary biliary cholangitis risk scores in a trial cohort of patients with primary biliary cholangitis

Brazilian society of hepatology recommendations for the diagnosis and management of autoimmune diseases of the liver

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