Published in:
22-08-2022 | Primary Biliary Cholangitis | Original Article
Response to Ursodeoxycholic Acid May Be Assessed Earlier to Allow Second-Line Therapy in Patients with Unresponsive Primary Biliary Cholangitis
Authors:
Guilherme Grossi Lopes Cançado, Cláudia Alves Couto, Debora Raquel Benedita Terrabuio, Eduardo Luiz Rachid Cançado, Cristiane Alves Villela-Nogueira, Maria Lucia Gomes Ferraz, Michelle Harriz Braga, Mateus Jorge Nardelli, Luciana Costa Faria, Nathalia Mota de Faria Gomes, Elze Maria Gomes Oliveira, Vivian Rotman, Maria Beatriz Oliveira, Simone Muniz Carvalho Fernandes da Cunha, Marlone Cunha-Silva, Liliana Sampaio Costa Mendes, Claudia Alexandra Pontes Ivantes, Liana Codes, Valéria Ferreira de Almeida e Borges, Fabio Heleno de Lima Pace, Mario Guimarães Pessoa, Laura Vilar Guedes, Izabelle Venturini Signorelli, Gabriela Perdomo Coral, Cynthia Levy, Paulo Lisboa Bittencourt, Members of the Brazilian Cholestasis Study Group Consortium
Published in:
Digestive Diseases and Sciences
|
Issue 2/2023
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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.