Development and Validation of a Scoring System to Predict Response to Obeticholic Acid in Primary Biliary Cholangitis.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
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De Vincentis Antonio, Ampuero Javier, Terracciani Francesca, D'Amato Daphne, Gerussi Alessio, Cristoferi Laura, Cazzagon Nora, Bonaiuto Emanuela, Floreani Annarosa, Calvaruso Vincenza, Cadamuro Luca, Degasperi Elisabetta, Morgando Anna, Vanni Ester, Lleo Ana, Colapietro Francesca, Alvaro Domenico, Castellaneta Antonino, Labanca Sara, Viganò Mauro, Distefano Marco, Pace Palitti Valeria, Ricci Chiara, De Matthaeis Nicoletta, Marzioni Marco, Gómez-Dominguez Elena, Montero Jose-Luis, Molina Esther, Garcia-Buey Luisa, Casado Marta, Berenguer Marina, Conde Isabel, Simon Miguel-Angel, Fuentes Javier, Costa-Moreira Pedro, Macedo Guilherme, Jorquera Francisco, Morillas Rosa-Maria, Presa Jose, Sousa Jose-Manuel, Gomes Dario, Santos Luis, Olveira Antonio, Hernandez-Guerra Manuel, Aburruza Leire, Santos Arsenio, Carvalho Armando, Uriz Juan, Gutierrez Maria-Luisa, Perez Elia, Chessa Luchino, Pellicelli Adriano, Marignani Massimo, Muratori Luigi, Niro Grazia Anna, Brunetto Maurizia, Ponziani Francesca Romana, Pompili Maurizio, Marra Fabio, Galli Andrea, Mussetto Alessandro, Alagna Giuliano, Simone Loredana, Bertino Gaetano, Rosina Floriano, Cozzolongo Raffaele, Russello Maurizio, Baiocchi Leonardo, Saitta Carlo, Terreni Natalia, Zolfino Teresa, Rigamonti Cristina, Vigano Raffaella, Cuccorese Giuseppe, Pozzoni Pietro, Pedone Claudio, Grasso Simone, Picardi Antonio, Invernizzi Pietro, Sacco Rodolfo, Izzi Antonio, Fernandez-Rodriguez Conrado, Vespasiani-Gentilucci Umberto, Carbone Marco, RECAPITULATE Investigators
Аннотация
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.