Rate of non-response to ursodeoxycholic acid in a large real-world cohort of primary biliary cholangitis patients in Italy

Vespasiani‐Gentilucci, Umberto; Rosina, F.; Pace‐Palitti, V.; Sacco, Rodolfo; Pellicelli, A.; Chessa, Luchino; Vincentis, Antonio De; Barlattani, M; Barlattani, A.; Feletti, Valentina; Mussetto, Alessandro; Zolfino, T.; Russello, Maurizio; Cozzolongo, Raffaele; Garrucciu, Giovanni; Niro, G.A.; Bacca, D.; Bertino, Gaetano; Claar, Ernesto; Ascione, Antonio; D’Adamo, Giuseppe; Adinolfi, Luigi Elio; Scifo, G.; Izzi, Antonio

doi:10.1080/00365521.2019.1669702

Cited by 12 publications

(8 citation statements)

References 32 publications

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“…The characteristics of our population, cared for by physicians who predominantly or exclusively practice hepatology and with good experience with PBC, was similar to those observed in recently published cohorts, in particular for age, gender ratio, and AMA positivity at presentation [8–13]. Results in the literature vary on the initial severity of disease.…”

Section: Discussionsupporting

confidence: 58%

Management of primary biliary cholangitis: results from a large real-life observational study in France and Belgium

Pariente¹,

Chazouillères

Causse

et al. 2020

European Journal of Gastroenterology &Amp; Hepatology

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Background and aims: To assess the characteristics, care, treatment response, and outcomes of primary biliary cholangitis (PBC) patients recently followed-up by hepato-gastroenterologists in various French and Belgian healthcare settings. Methods: This retrospective cohort study included patients with PBC who recently visited 79 hepato-gastroenterologists in France and Belgium. Data were collected at the time of diagnosis and at last visit and were compared according to biochemical response (BR) to ursodeoxycholic acid (UDCA) (BR), using Paris I–II criteria, and clinical outcomes. Results: A total of 436 patients (mean age at diagnosis 57 years, 88% females, median follow-up 5.2 years) were included. Liver biopsy, transient elastography, or none of these two procedures were performed at baseline in 216 (50%), 194 (45%), and 107 (25%) patients, respectively. Late-stage disease (histological stage III or IV, or transient elastography ≥9.6 kPa, or bilirubin >17 µM and albumin <35 g/L, or platelets <150.000/µl, or unequivocal signs of portal hypertension or cirrhosis) was reported in 37% of patients. UDCA was taken by 95% of patients (27% had suboptimal dosage). Inadequate BR was observed in 37% of patients. Clinicians overestimated disease control. Liver-related complications occurred in 9% of patients. Bilirubin and albumin independently predicted inadequate BR; advanced disease stage and inadequate BR independently predicted complications. Conclusions: Recently followed-up French and Belgian patients with PBC had homogeneous management. Late stage at diagnosis and inadequate BR were reported in around 40% of patients. Disease control was frequently overestimated by clinicians. Disease stage and BR were the main prognostic factors.

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Section: Discussionsupporting

confidence: 58%

Management of primary biliary cholangitis: results from a large real-life observational study in France and Belgium

Pariente¹,

Chazouillères

Causse

et al. 2020

European Journal of Gastroenterology &Amp; Hepatology

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“…In addition, when assessing response rates according to the Toronto criterion, we found a non-response rate of 28.1%, while a recently published German study found a non-response rate of only 16.2% according to this criterion [ 22 ]. These higher percentages of UDCA non-response in our study could be attributed to the accumulation of difficult to treat patients in a referral tertiary center [ 17 ].…”

Section: Discussionmentioning

confidence: 96%

“…Recently, a latitudinal geoepidemiological pattern of occurrence has been proposed, with a higher prevalence in northern countries [ 16 ]. Interestingly, the rates of non-response to UDCA also varied in different studies depending on the geographical region and the study population [ 17 ]. For example, the inadequate response rate according to the Toronto criteria were found to be as high as 38.6% in a single center in England and 33% in a population study in Iceland [ 18 , 19 ], while in a single center in China and in a multicenter study in Italy, lower non-responder rates of 17% and 20.1%, were reported, respectively [ 17 , 20 ].…”

Section: Introductionmentioning

confidence: 99%

Real-World Management of Patients with Primary Biliary Cholangitis—A Retrospective Study from a Tertiary Medical Center in Israel

Yehezkel

Israel

Houri

et al. 2021

JCM

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Background: Primary biliary cholangitis (PBC) is a rare autoimmune liver disease with variation in prevalence, phenotype and prognosis across different geographical regions. Little is known about PBC in Israel. Our aim was to characterize the demography, clinical presentation, treatment patterns and prognosis in a cohort of PBC patients followed in a referral center in central Israel. Methods: Clinical, demographic and laboratory data were collected from the medical records of PBC patients followed at Tel Aviv Medical Center in the years 2003–2020. Results: We have identified 189 patients with a confirmed diagnosis of PBC; 92.6% were female and the mean age at diagnosis was 54.7 years. Thirty-nine percent were diagnosed with another autoimmune disease and 5.9% were diagnosed with a PBC-AIH (autoimmune hepatitis) variant syndrome. Ninety-six percent were treated with ursodeoxycholic acid (UDCA) at a mean dose of 13.3 mg/kg. A total of 28.1% were found with inadequate response to UDCA according to the Toronto criteria, and 53% of the UDCA non-responders were treated with bezafibrate. Younger age at diagnosis, higher baseline levels of alkaline phosphatase (ALP) and gamma-glutamyltransferase (GGT), AIH-PBC variant and positive anti-smooth muscle antibodies (ASMA) were associated with an inadequate UDCA response. In a multivariable analysis, higher ALP at diagnosis (OR = 1.92 CI 1.11–3.20 per 50-unit change, p = 0.018) and ASMA (OR = 27.6 CI 2.58–295, p = 0.006) independently predicted inadequate UDCA response. Higher alanine transaminase (ALT), ALP and GGT, lower albumin, younger age at diagnosis and pruritus conferred an increased risk for disease progression. Conclusions: Disease characteristics, treatment patterns, response to therapy and prognosis of a PBC patient cohort in a tertiary center in central Israel were revealed. The results highlight the importance of risk stratification in PBC, specifically in younger patients, those presenting with a high level of liver enzymes and in ASMA-positive patients with an assumed diagnosis of the AIH-PBC variant.

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“…Primary biliary cholangitis (PBC) is an autoimmune disease of the small‐ and medium‐size bile ducts causing chronic cholestasis, which, if untreated or undertreated, can slowly progress to liver fibrosis and cirrhosis 1 . Ursodeoxycholic acid (UDCA) is the first‐line treatment and is effective in ~60% of patients, depending on the definition of treatment response applied 2–4 . Obeticholic acid (OCA) is the registered second‐line treatment which is offered to patients who do not achieve a satisfactory response, or are intolerant, to UDCA 1 .…”

Section: Introductionmentioning

confidence: 99%

“… 1 Ursodeoxycholic acid (UDCA) is the first‐line treatment and is effective in ~60% of patients, depending on the definition of treatment response applied. 2 , 3 , 4 Obeticholic acid (OCA) is the registered second‐line treatment which is offered to patients who do not achieve a satisfactory response, or are intolerant, to UDCA. 1 The addition of OCA can rescue to response ~40% of UDCA non‐responders.…”

Section: Introductionmentioning

confidence: 99%

Predictors of serious adverse events and non‐response in cirrhotic patients with primary biliary cholangitis treated with obeticholic acid

Vincentis

D’Amato

Cristoferi

et al. 2022

Liver International

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Background & Aims: Obeticholic acid (OCA) has recently been restricted in patients with primary biliary cholangitis (PBC) with "advanced cirrhosis" because of its narrow therapeutic index. We aimed to better define the predicting factors of hepatic serious adverse events (SAEs) and non-response in cirrhotic patients undergoing OCA therapy.Methods: Safety and efficacy of treatment were evaluated in a cohort of consecutive PBC cirrhotic patients started with OCA. OCA response was evaluated according to the Poise criteria. Risk factors for hepatic SAEs and non-response were reported as risk ratios (RR) with 95% confidence intervals (CIs).Results: One hundred PBC cirrhotics were included, 97 Child-Pugh class A and 3 class B. Thirty-one had oesophageal varices and 5 had a history of ascites. Thirty-three per cent and 32% of patients achieved a biochemical response at 6 and 12 months respectively. Male sex (adjusted-RR 1.75, 95%CI 1.42-2.12), INR (1.37, 1.00-1.87),

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Rate of non-response to ursodeoxycholic acid in a large real-world cohort of primary biliary cholangitis patients in Italy

Cited by 12 publications

References 32 publications

Management of primary biliary cholangitis: results from a large real-life observational study in France and Belgium

Management of primary biliary cholangitis: results from a large real-life observational study in France and Belgium

Real-World Management of Patients with Primary Biliary Cholangitis—A Retrospective Study from a Tertiary Medical Center in Israel

Predictors of serious adverse events and non‐response in cirrhotic patients with primary biliary cholangitis treated with obeticholic acid

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