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. 2024 Oct;22(10):2062-2074.e11.
doi: 10.1016/j.cgh.2024.05.008. Epub 2024 May 22.

Development and Validation of a Scoring System to Predict Response to Obeticholic Acid in Primary Biliary Cholangitis

Antonio De Vincentis  1 , Javier Ampuero  2 , Francesca Terracciani  3 , Daphne D'Amato  4 , Alessio Gerussi  5 , Laura Cristoferi  5 , Nora Cazzagon  6 , Emanuela Bonaiuto  6 , Annarosa Floreani  6 , Vincenza Calvaruso  7 , Luca Cadamuro  7 , Elisabetta Degasperi  8 , Anna Morgando  4 , Ester Vanni  4 , Ana Lleo  9 , Francesca Colapietro  9 , Domenico Alvaro  10 , Antonino Castellaneta  11 , Sara Labanca  12 , Mauro Viganò  13 , Marco Distefano  14 , Valeria Pace Palitti  15 , Chiara Ricci  16 , Nicoletta De Matthaeis  17 , Marco Marzioni  18 , Elena Gómez-Dominguez  19 , Jose-Luis Montero  20 , Esther Molina  21 , Luisa Garcia-Buey  22 , Marta Casado  23 , Marina Berenguer  24 , Isabel Conde  24 , Miguel-Angel Simon  25 , Javier Fuentes  26 , Pedro Costa-Moreira  27 , Guilherme Macedo  27 , Francisco Jorquera  28 , Rosa-Maria Morillas  29 , Jose Presa  30 , Jose-Manuel Sousa  2 , Dario Gomes  31 , Luis Santos  31 , Antonio Olveira  32 , Manuel Hernandez-Guerra  33 , Leire Aburruza  34 , Arsenio Santos  35 , Armando Carvalho  35 , Juan Uriz  36 , Maria-Luisa Gutierrez  37 , Elia Perez  37 , Luchino Chessa  38 , Adriano Pellicelli  39 , Massimo Marignani  40 , Luigi Muratori  41 , Grazia Anna Niro  42 , Maurizia Brunetto  43 , Francesca Romana Ponziani  44 , Maurizio Pompili  44 , Fabio Marra  45 , Andrea Galli  45 , Alessandro Mussetto  46 , Giuliano Alagna  47 , Loredana Simone  48 , Gaetano Bertino  49 , Floriano Rosina  50 , Raffaele Cozzolongo  51 , Maurizio Russello  52 , Leonardo Baiocchi  53 , Carlo Saitta  54 , Natalia Terreni  55 , Teresa Zolfino  56 , Cristina Rigamonti  57 , Raffaella Vigano  58 , Giuseppe Cuccorese  59 , Pietro Pozzoni  60 , Claudio Pedone  61 , Simone Grasso  62 , Antonio Picardi  3 , Pietro Invernizzi  5 , Rodolfo Sacco  63 , Antonio Izzi  64 , Conrado Fernandez-Rodriguez  65 , Umberto Vespasiani-Gentilucci  66 , Marco Carbone  67 ; RECAPITULATE Investigators
Collaborators, Affiliations

Development and Validation of a Scoring System to Predict Response to Obeticholic Acid in Primary Biliary Cholangitis

Antonio De Vincentis et al. Clin Gastroenterol Hepatol. 2024 Oct.

Erratum in

  • Correction.
    [No authors listed] [No authors listed] Clin Gastroenterol Hepatol. 2025 Feb;23(2):381. doi: 10.1016/j.cgh.2024.11.001. Epub 2024 Dec 8. Clin Gastroenterol Hepatol. 2025. PMID: 39652020 No abstract available.

Abstract

Background & aims: Obeticholic acid (OCA) is the only licensed second-line therapy for primary biliary cholangitis (PBC). With novel therapeutics in advanced development, clinical tools are needed to tailor the treatment algorithm. We aimed to derive and externally validate the OCA response score (ORS) for predicting the response probability of individuals with PBC to OCA.

Methods: We used data from the Italian RECAPITULATE (N = 441) and the IBER-PBC (N = 244) OCA real-world prospective cohorts to derive/validate a score including widely available variables obtained either pre-treatment (ORS) or also after 6 months of treatment (ORS+). Multivariable Cox regressions with backward selection were applied to obtain parsimonious predictive models. The predicted outcomes were biochemical response according to POISE (alkaline phosphatase [ALP]/upper limit of normal [ULN]<1.67 with a reduction of at least 15%, and normal bilirubin), or ALP/ULN<1.67, or normal range criteria (NR: normal ALP, alanine aminotransferase [ALT], and bilirubin) up to 24 months.

Results: Depending on the response criteria, ORS included age, pruritus, cirrhosis, ALP/ULN, ALT/ULN, GGT/ULN, and bilirubin. ORS+ also included ALP/ULN and bilirubin after 6 months of OCA therapy. Internally validated c-statistics for ORS were 0.75, 0.78, and 0.72 for POISE, ALP/ULN<1.67, and NR response, which raised to 0.83, 0.88, and 0.81 with ORS+, respectively. The respective performances in validation were 0.70, 0.72, and 0.71 for ORS and 0.80, 0.84, and 0.78 for ORS+. Results were consistent across groups with mild/severe disease.

Conclusions: We developed and externally validated a scoring system capable to predict OCA response according to different criteria. This tool will enhance a stratified second-line therapy model to streamline standard care and trial delivery in PBC.

Keywords: Obeticholic Acid; Predictive Model; Primary Biliary Cholangitis.

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