Effectiveness and safety of obeticholic acid in a Southern European multicenter cohort of patients with primary biliary cholangitis and suboptimal response to ursodeoxycholic acid
Por:
Gomez, E, Buey, LG, Molina, E, Casado, M, Conde, I, Berenguer, M, Jorquera, F, Simon, MA, Olveira, A, Hernandez-Guerra, M, Mesquita, M, Presa, J, Costa-Moreira, P, Macedo, G, Arenas, JI, Sousa, JM, Ampuero, J, Morillas, RM, Santos, A, De Carvalho, A, Uriz, J, Carrion, JA, Gutierrez, ML, Perez-Fernandez, E and Fernandez-Rodriguez, CM
Publicada:
1 feb 2021
Ahead of Print:
1 dic 2020
Resumen:
Background Obeticholic acid (OCA) was recently approved as the only on-label alternative for patients with primary biliary cholangitis (PBC) with intolerance or suboptimal response to ursodeoxycholic acid (UDCA). However, few data are available outside clinical trials.
Aim To assess the effectiveness and safety of OCA in a real-world cohort of patients with non-effective UDCA therapy.
Methods Open-label, prospective, real-world, multicentre study, enrolling consecutive patients who did not meet Paris II criteria, from 18 institutions in Spain and Portugal. Effectiveness was assessed by the changes in GLOBE and UK-PBC scores from baseline. POISE and Paris II criteria were evaluated after 12 months of OCA . Liver fibrosis was evaluated by FIB-4 and AST to platelet ratio index (APRI).
Results One hundred and twenty patients were eligible, median time since PBC diagnosis 9.3 (4.0-13.8) years, 21.7% had cirrhosis, and 26.7% received had previous or concomitant treatment with fibrates. Seventy-eight patients completed at least 1 year of OCA. The Globe-PBC score decreased to 0.17 (95% CI 0.05 to 0.28; P = 0.005) and the UK-PBC score decreased to 0.81 (95% CI -0.19 to 1.80; P = 0.11). There was a significant decrease in alkaline phosphatase of 81.3 U/L (95% CI 42.5 to 120; P < 0.001), ALT 22.1 U/L (95% CI 10.4 to 33.8; P < 0.001) and bilirubin 0.12 mg/dL (95% CI 0 to 0.24; P = 0.044). FIB-4 and APRI remained stable. According to the POISE criteria, 29.5% (23 out of 78) achieved response. The adverse events rate was 35%; 11.67% discontinued (8.3% due to pruritus).
Conclusions This study supports data from phase III trials with significant improvement of PBC-Globe continuous prognostic marker score among OCA-treated patients with good tolerability.
Filiaciones:
Gomez, E:
Hosp Univ 12 Octubre, Madrid, Spain
Buey, LG:
Hosp Univ La Princesa, Madrid, Spain
Molina, E:
Complexo Hosp Univ Santiago, Santiago, Spain
Casado, M:
Complexo Hosp Univ Santiago, Santiago, Spain
Conde, I:
Hosp Univ Torrecardenas, Almeria, Spain
Berenguer, M:
Hosp Univ Torrecardenas, Almeria, Spain
Jorquera, F:
Hosp Univ La Fe, Valencia, Spain
CIBEREHD, Valencia, Spain
Simon, MA:
Complejo Asistencial Univ Leon Ciberehd, Leon, Spain
Olveira, A:
Hosp Univ Lozano Biesa, Zaragoza, Spain
Hernandez-Guerra, M:
Hosp Univ La Paz, Madrid, Spain
Mesquita, M:
Ctr Hosp Tras Os Montes & Alto Douro, Vila Real, Portugal
Presa, J:
Ctr Hosp Sao Joao, Porto, Portugal
Costa-Moreira, P:
Hosp Univ Canarias, Santa Cruz De Tenerife, Spain
Macedo, G:
Hosp Univ Canarias, Santa Cruz De Tenerife, Spain
Arenas, JI:
Hosp Univ Donostia, San Sebastian, Spain
Sousa, JM:
Hosp Virgen Rocio Ciberehd, Seville, Spain
Ampuero, J:
Hosp Badalona Germans Trias & Pujol, Barcelona, Spain
:
Hosp Badalona Germans Trias & Pujol, Barcelona, Spain
Santos, A:
Ctr Hosp Univ Coimbra, Coimbra, Spain
De Carvalho, A:
Ctr Hosp Univ Coimbra, Coimbra, Spain
Uriz, J:
Complejo Hosp Navarra, Navarra, Spain
Carrion, JA:
Hosp Mar, Barcelona, Spain
Gutierrez, ML:
Hosp Univ Fdn Alcorcon, Madrid, Spain
Perez-Fernandez, E:
Hosp Univ Fdn Alcorcon, Madrid, Spain
Fernandez-Rodriguez, CM:
Hosp Univ Fdn Alcorcon, Madrid, Spain
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