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

• Published in: Digestive diseases and sciences Vol. 68; no. 2; pp. 514 - 520
• Main Authors: Cançado, Guilherme Grossi Lopes, Couto, Cláudia Alves, Terrabuio, Debora Raquel Benedita, Cançado, Eduardo Luiz Rachid, Villela-Nogueira, Cristiane Alves, Ferraz, Maria Lucia Gomes, Braga, Michelle Harriz, Nardelli, Mateus Jorge, Faria, Luciana Costa, de Faria Gomes, Nathalia Mota, Oliveira, Elze Maria Gomes, Rotman, Vivian, Oliveira, Maria Beatriz, da Cunha, Simone Muniz Carvalho Fernandes, Cunha-Silva, Marlone, Mendes, Liliana Sampaio Costa, Ivantes, Claudia Alexandra Pontes, Codes, Liana, de Almeida e Borges, Valéria Ferreira, de Lima Pace, Fabio Heleno, Pessoa, Mario Guimarães, Guedes, Laura Vilar, Signorelli, Izabelle Venturini, Coral, Gabriela Perdomo, Levy, Cynthia, Bittencourt, Paulo Lisboa
• Format: Journal Article
• Language: English
• Published: New York Springer US 01.02.2023; Springer; Springer Nature B.V
• Subjects: Acids; Adult; Aged; Antibodies; Biochemistry; Cholagogues and Choleretics - therapeutic use; Cholangitis; Consortia; Drug dosages; Enzymes; Female; Gallbladder diseases; Gastroenterology; Hepatology; Histology; Humans; Laboratories; Liver; Liver cirrhosis; Liver Cirrhosis, Biliary - drug therapy; Liver diseases; Male; Medical prognosis; Medicine; Medicine & Public Health; Middle Aged; Oncology; Original Article; Patient compliance; Phosphatase; Phosphatases; Retrospective Studies; Statistical analysis; Transplant Surgery; Transplants & implants; Ursodeoxycholic Acid - therapeutic use; Ursodiol; Variables; Primary biliary cholangitis; Treatment; Second-line therapy; Ursodeoxycholic acid
• ISSN: 0163-2116; 1573-2568; 1573-2568
• DOI: 10.1007/s10620-022-07654-x

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.