Alkaline phosphatase and liver fibrosis at diagnosis are associated with deep response to ursodeoxycholic acid in primary biliary cholangitis - 24/08/24
, Patrícia da Silva Fucuta c, d, Nathalia Mota de Faria Gomes e, Cláudia Alves Couto a, Eduardo Luiz Rachid Cançado f, Debora Raquel Benedita Terrabuio f, Cristiane Alves Villela‑Nogueira g, Michelle Harriz Braga f, Mateus Jorge Nardelli a, Luciana Costa Faria a, Elze Maria Gomes Oliveira h, Vivian Rotman g, Maria Beatriz Oliveira i, Simone Muniz Carvalho Fernandes da Cunha j, Marlone Cunha da Silva k, Liliana Sampaio Costa Mendes l, Claudia Alexandra Pontes Ivantes m, Liana Codes n, o, Valéria Ferreira de Almeida e Borges p, q, Fabio Heleno de Lima Pace r, Mário Guimarães Pessôa f, Izabelle Venturini Signorelli s, Gabriela Perdomo Coral t, Paulo Lisboa Bittencourt n, o, Maria Lucia Gomes Ferraz eHighlights |
• | Nomalization of alkaline phosphatase and bilirubin, defined as deep response, is a new treatment target for PBC. |
• | Cirrhosis at diagnosis and elevated baseline alkaline phosphatase levels were associated with decreased likelihood of achieving a deep response to UDCA. |
• | The UDCA response score is inadequate for predicting deep response in the Brazilian PBC population. |
Abstract |
Objective |
Primary biliary cholangitis is a chronic and progressive autoimmune liver disease, whose prognosis can be improved by normalizing alkaline phosphatase and bilirubin. While ursodeoxycholic acid (UDCA) is first line standard of care, approximately 40 % of patients exhibit incomplete response. We aimed to identify prognostic markers for deep response to UDCA therapy at presentation.
Patient and methods |
Data from the Brazilian Cholestasis Study Group cohort were analyzed retrospectively. Patients were assessed for deep response, defined as normal alkaline phosphatase and bilirubin, after 1 year of UDCA treatment. Additionally, the performance of the UDCA response score in predicting deep response was evaluated.
Results |
A total of 297 patients were analyzed, with 57.2 % achieving an adequate response according to the Toronto criteria, while 22.9 % reached deep response. Cirrhosis (OR 0.460; 95 % CI 0.225–0.942; p = 0.034) and elevated baseline alkaline phosphatase levels (OR 0.629; 95 % CI 0.513–0.770; p < 0.001) were associated with reduced odds of deep response. The UDCA response score exhibited moderate discrimination power (AUROC = 0.769) but lacked calibration.
Conclusions |
Baseline ALP and liver fibrosis emerge as the most important prognostic factors to predict normalization of alkaline phosphatase and bilirubin after UDCA. The UDCA response score was inadequate for predicting deep response in the Brazilian PBC population.
Le texte complet de cet article est disponible en PDF.Graphical abstract |
Keywords : Biochemical response, Biochemical remission, Prognosis, Ursodeoxycholic acid, Predictive score, Risk stratification, Deep response
Abbreviations : UDCA, PBC, URS, AMA, ALP, ULN, AST, ALT, GGT, TB
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Vol 48 - N° 8
Article 102453- octobre 2024 Retour au numéro
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