Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis

Harms, Maren H.; Veer, Rozanne C de; Lammers, Willem J; Corpechot, Christophe; Thorburn, Douglas; Janssen, Harry L A; Lindor, Keith D.; Trivedi, Palak; Hirschfield, Gideon; Pares, A.; Floreani, Annarosa; Mayo, Marlyn J.; Invernizzi, Pietro; Battezzati, Pier Maria; Nevens, Frederik; Ponsioen, Cyriel Y.; Mason, Andrew L.; Kowdley, Kris V.; Hansen, Bettina E.; Buuren, Henk R. van; Meer, Adriaan J. van der

doi:10.1136/gutjnl-2019-319057

Cited by 30 publications

(16 citation statements)

References 29 publications

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“…We extend this observation with comparisons with the survival of the general population, as well as the subgroup of patients with the best predicted outcome (those with low GLOBE scores). Still, the prognosis of patients with a low GLOBE score can be considered good irrespective of the ALP level, so that the absolute survival gain of add‐on therapy may be modest 22 . Especially for young patients, however, optimised outcome may be preferred 24 .…”

Section: Discussionmentioning

confidence: 99%

Liver transplant‐free survival according to alkaline phosphatase and GLOBE score in patients with primary biliary cholangitis treated with ursodeoxycholic acid

Veer

Harms

Corpechot

et al. 2022

Aliment Pharmacol Ther

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Background: After 1 year of ursodeoxycholic acid (UDCA), patients with primary biliary cholangitis (PBC) may have a normal GLOBE score despite high alkaline phosphatase (ALP) levels. Aim:To assess the association between ALP and liver transplantation (LT)-free survival according to the GLOBE score Methods: Among patients with a normal or elevated GLOBE score in the Global PBC cohort, the association between ALP after 1 year of UDCA and the risk of LT/death was assessed. The LT-free survival was compared with that of a matched general population.Results: After 1 year of UDCA, ALP was associated with the risk of LT/death (aHR 1.31, 95% CI 1.003-1.72, p = 0.048) among 2729 patients with a normal GLOBE score. The 10-year LT-free survival among these patients with an ALP >2.0 × ULN was 94.0% (95% CI 90.1-97.9) for those <50 years, and 82.6% (95% CI 76.5-88.7) for those ≥50 years, which was significantly lower (p = 0.040) and similar (p = 0.736) to that of the matched population, respectively. The 10-year LT-free survival in patients ≥50 years with normal GLOBE score and normal ALP (90.8%, 95% CI 87.7-93.9) was significantly higher (p = 0.022) than the matched population. Among 1045 patients with an elevated GLOBE score, ALP was associated with LT/death only in those <50 years (aHR 1.38, 95% CI 1.06-1.81, p = 0.016). Conclusion:The LT-free survival of patients with PBC with a normal GLOBE score is optimal in case of normal ALP levels, also in relation to the general population. Despite their generally favourable prognosis, an elevated ALP level may still indicate a need for add-on therapy. How to cite this article: de Veer RC, Harms MH, Corpechot C, Thorburn D, Invernizzi P, Janssen HLA, et al. Liver transplant-free survival according to alkaline phosphatase and GLOBE score in patients with primary biliary cholangitis treated with ursodeoxycholic acid.

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Section: Discussionmentioning

confidence: 99%

Liver transplant‐free survival according to alkaline phosphatase and GLOBE score in patients with primary biliary cholangitis treated with ursodeoxycholic acid

Veer

Harms

Corpechot

et al. 2022

Aliment Pharmacol Ther

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“…60-80 % ein suffizientes laborchemisches Ansprechen [12]. Neben der Verbesserung laborchemischer Parameter hilft die Dauertherapie mit UDCA effektiv, klinisch relevante Endpunkte (Lebertransplantation und leberbezogener Tod) zu verhindern [13]. Bereits die Therapie von 11 Patient*innen über die Zeit von 5 Jahren kann im Mittel einen Todesfall oder eine Transplantation verhindern [13].…”

Section: Ursodesoxycholsäure (Udca)unclassified

“…Neben der Verbesserung laborchemischer Parameter hilft die Dauertherapie mit UDCA effektiv, klinisch relevante Endpunkte (Lebertransplantation und leberbezogener Tod) zu verhindern [13]. Bereits die Therapie von 11 Patient*innen über die Zeit von 5 Jahren kann im Mittel einen Todesfall oder eine Transplantation verhindern [13]. Um das biochemische Ansprechen zu definieren, wurden seit Etablierung der UDCA-Therapie wiederholt unterschiedliche Kriterien definiert (Barcelona, Paris I und II, Rotterdam, Toronto-Kriterien).…”

Section: Ursodesoxycholsäure (Udca)unclassified

Autoimmune Lebererkrankungen – ein Update (Teil 2)

Schregel¹,

Steinmann²,

Schramm³

2022

Gastroenterologie up2date

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“…The necessity to promptly initiate UDCA treatment is strong, as the number needed to treat to prevent one liver transplant or death within 5 years is 11 62 . UDCA is a naturally occurring hydrophilic bile acid affecting the enterohepatic cycle and cholestasis, working by decreasing toxic effects of bile acids to BEC membranes, inhibiting intestinal absorption of bile acids, increasing biliary secretion of bile acids, increasing elimination of toxic substances, stimulating cholangiocytes to secrete bicarbonate‐rich fluid, and augmenting micelle formation 63 .…”

Section: What Is First‐line Treatment For Pbc?mentioning

confidence: 99%

Review article: pathophysiology and management of primary biliary cholangitis

Leung

Deeb

Hirschfield

2020

Aliment Pharmacol Ther

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SummaryBackgroundPrimary biliary cholangitis (PBC), an immune‐mediated disease characterised by destruction of intrahepatic bile ducts, results in progressive damage to the biliary tree, cholestasis and ultimately advanced liver disease. In the last decade, advances in practice have improved clinical care, driven novel therapeutic options and improved risk stratification tools.AimsTo provide an overview of the disease characteristics of PBC and review a patient‐centred management approach for the clinical team caring for those with PBC.MethodsWe reviewed the current literature and guidelines on PBC with a focus on management and therapies.ResultsA confident diagnosis of PBC is usually made based on serum liver tests and immune serology. Management of PBC should focus on three main ‘process’ pillars: (a) treat and risk‐stratify through use of biochemical and prognostic criteria; (b) manage concurrent symptoms and other associated diseases; and (c) stage disease, monitor progression and prevent complications. With ongoing complexities in management, including a newly licensed therapy (obeticholic acid) and alternative non‐licensed treatments and ongoing clinical trials, discussion with PBC expert centres is encouraged.ConclusionsPBC is a dynamic disease wherein current treatment goals have become appropriately ambitious. Goals of care should prioritise prevention of end‐stage liver disease and amelioration of patient symptom burden for all.

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Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis

Cited by 30 publications

References 29 publications

Liver transplant‐free survival according to alkaline phosphatase and GLOBE score in patients with primary biliary cholangitis treated with ursodeoxycholic acid

Liver transplant‐free survival according to alkaline phosphatase and GLOBE score in patients with primary biliary cholangitis treated with ursodeoxycholic acid

Autoimmune Lebererkrankungen – ein Update (Teil 2)

Review article: pathophysiology and management of primary biliary cholangitis

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