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Multicenter Study
. 2023 Feb;68(2):514-520.
doi: 10.1007/s10620-022-07654-x. Epub 2022 Aug 22.

Response to Ursodeoxycholic Acid May Be Assessed Earlier to Allow Second-Line Therapy in Patients with Unresponsive Primary Biliary Cholangitis

Guilherme Grossi Lopes Cançado  1   2 , Cláudia Alves Couto  3 , Debora Raquel Benedita Terrabuio  4 , Eduardo Luiz Rachid Cançado  4 , Cristiane Alves Villela-Nogueira  5 , Maria Lucia Gomes Ferraz  6 , Michelle Harriz Braga  4 , Mateus Jorge Nardelli  3 , Luciana Costa Faria  3 , Nathalia Mota de Faria Gomes  6 , Elze Maria Gomes Oliveira  7 , Vivian Rotman  5 , Maria Beatriz Oliveira  8 , Simone Muniz Carvalho Fernandes da Cunha  9 , Marlone Cunha-Silva  10 , Liliana Sampaio Costa Mendes  11 , Claudia Alexandra Pontes Ivantes  12 , Liana Codes  9   13 , Valéria Ferreira de Almeida E Borges  14   15 , Fabio Heleno de Lima Pace  16 , Mario Guimarães Pessoa  4 , Laura Vilar Guedes  4 , Izabelle Venturini Signorelli  17 , Gabriela Perdomo Coral  18 , Cynthia Levy  19 , Paulo Lisboa Bittencourt  13   20 ; Members of the Brazilian Cholestasis Study Group Consortium
Affiliations
Multicenter Study

Response to Ursodeoxycholic Acid May Be Assessed Earlier to Allow Second-Line Therapy in Patients with Unresponsive Primary Biliary Cholangitis

Guilherme Grossi Lopes Cançado et al. Dig Dis Sci. 2023 Feb.

Abstract

Background: Response to ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC) has been traditionally assessed 1 to 2 years after treatment initiation. With the development of new drugs, some patients may benefit from an earlier introduction of second-line therapies.

Aims: This study aims to identify whether well-validated response criteria could correctly identify individuals likely to benefit from add-on second-line therapy at 6 months.

Methods: Analysis of a multicenter retrospective cohort which included only patients with clear-cut PBC.

Results: 206 patients with PBC (96.6% women; mean age 54 ± 12 years) were included. Kappa concordance was substantial for Toronto (0.67), Rotterdam (0.65), Paris 1 (0.63) and 2 (0.63) criteria at 6 and 12 months, whereas Barcelona (0.47) and POISE trial (0.59) criteria exhibited moderate agreement. Non-response rates to UDCA was not statistically different when assessed either at 6 or 12 months using Toronto, Rotterdam or Paris 2 criteria. Those differences were even smaller or absent in those subjects with advanced PBC. Mean baseline alkaline phosphatase was 2.73 ± 1.95 times the upper limit of normal (× ULN) among responders versus 5.05 ± 3.08 × ULN in non-responders (p < 0.001).

Conclusions: After 6 months of treatment with UDCA, the absence of response by different criteria could properly identify patients who could benefit from early addition of second-line therapies, especially in patients with advanced disease or high baseline liver enzymes levels.

Keywords: Primary biliary cholangitis; Second-line therapy; Treatment; Ursodeoxycholic acid.

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References

    1. Corpechot C, Carrat F, Bonnand AM et al. The effect of ursodeoxycholic acid therapy on liver fibrosis progression in primary biliary cirrhosis. Hepatology. 2000;32:1196–1199. - DOI
    1. Corpechot C, Abenavoli L, Rabahi N et al. Biochemical response to ursodeoxycholic acid and long-term prognosis in primary biliary cirrhosis. Hepatology. 2008;48:871–877. - DOI
    1. Harms MH, van Buuren HR, Corpechot C et al. Ursodeoxycholic acid therapy and liver transplant-free survival in patients with primary biliary cholangitis. J Hepatol. 2019;71:357–365. - DOI
    1. Lammers WJ, van Buuren HR, Hirschfield GM et al. Levels of alkaline phosphatase and bilirubin are surrogate end points of outcomes of patients with primary biliary cirrhosis: an international follow-up study. Gastroenterology. 2014;147:1338-1349.e5. - DOI
    1. Itakura J, Izumi N, Nishimura Y et al. Prospective randomized crossover trial of combination therapy with bezafibrate and UDCA for primary biliary cirrhosis. Hepatol Res. 2004;29:216–222. - DOI

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