Longitudinal outcomes of obeticholic acid therapy in ursodiol-nonresponsive primary biliary cholangitis: Stratifying the impact of add-on fibrates in real-world practice
Por:
Gómez, E, Montero, JL, Molina, E, García-Buey, L, Casado, M, Fuentes, J, Simón, MA, Díaz-González, A, Jorquera, F, Morillas, RM, Presa, J, Berenguer, M, Conde, MI, Olveira, A, Macedo, G, Garrido, I, Hernández-Guerra, M, Olivas, I, Rodríguez-Taje, S, Londoño, M, Sousa, JM, Ampuero, J, Romero-González, E, González-Padilla, S, Escudero-García, D, Carvalho, A, Santos, A, Gutiérrez, ML, Pérez-Fernández, E, Aburruza, L, Uriz, J, Gomes, D, Santos, L, Martínez-González, J, Albillos, A and Fernández-Rodríguez, CM
Publicada:
1 jun 2024
Ahead of Print:
1 may 2024
Resumen:
Background: Suboptimal response to ursodeoxycholic acid occurs in 40% of primary biliary cholangitis (PBC) patients, affecting survival. Achieving a deep response (normalisation of alkaline phosphatase [ALP] and bilirubin <= 0.6 upper limit of normal) improves survival. Yet, the long-term effectiveness of second-line treatments remains uncertain. Aims: To evaluate the long-term effectiveness of obeticholic acid (OCA) +/- fibrates. Focusing on biochemical response (ALP <= 1.67 times the upper limit of normal, with a decrease of at least 15% from baseline and normal bilirubin levels), normalisation of ALP, deep response and biochemical remission (deep response plus aminotransferase normalisation). Methods: We conducted a longitudinal, observational, multicentre study involving ursodeoxyccholic acid non-responsive PBC patients (Paris-II criteria) from Spain and Portugal who received OCA +/- fibrates. Results: Of 255 patients, median follow-up was 35.1 months (IQR: 20.2-53). The biochemical response in the whole cohort was 47.2%, 61.4% and 68.6% at 12, 24 and 36 months. GLOBE-PBC and 5-year UK-PBC scores improved (p < 0.001). Triple therapy (ursodeoxycholic acid plus OCA plus fibrates) had significantly higher response rates than dual therapy (p = 0.001), including ALP normalisation, deep response and biochemical remission (p < 0.001). In multivariate analysis, triple therapy remained independently associated with biochemical response (p = 0.024), alkaline phosphatase normalisation, deep response and biochemical remission (p < 0.001). Adverse effects occurred in 41.2% of cases, leading to 18.8% discontinuing OCA. Out of 55 patients with cirrhosis, 12 developed decompensation. All with baseline portal hypertension. Conclusion: Triple therapy was superior in achieving therapeutic goals in UDCA-nonresponsive PBC. Decompensation was linked to pre-existing portal hypertension.
Filiaciones:
Gómez, E:
Hosp Univ 12 Octubre, Madrid, Spain
Montero, JL:
Hosp Univ Reina Sofia, Cordoba, Spain
Molina, E:
Complexo Hosp Univ Santiago, Coruna, Spain
García-Buey, L:
Hosp Univ La Princesa, Madrid, Spain
Casado, M:
Hosp Univ Torrecardenas, Almeria, Spain
Fuentes, J:
Hosp Univ Miguel Servet, Zaragoza, Spain
Simón, MA:
Hosp Clin Univ Lozano Blesa, Zaragoza, Spain
Univ Zaragoza, Zaragoza, Spain
Díaz-González, A:
Hosp Univ Marques Valdecilla, Santander, Spain
Jorquera, F:
Complejo Hosp Leon, Leon, Spain
:
Hosp Badalona Germans Trias & Pujol, Badalona, Spain
Presa, J:
Ctr Hosp Tras Os Montes Alto Douro, Vila Real, Portugal
Berenguer, M:
Hosp Univ La Fe, Valencia, Spain
Univ Valencia, Valencia, Spain
Conde, MI:
Hosp Univ La Fe, Valencia, Spain
Olveira, A:
Hosp Univ La Paz, Madrid, Spain
Macedo, G:
CHU Sao Joao CHUSJ, Serv Gastrenterol, Porto, Portugal
Garrido, I:
CHU Sao Joao CHUSJ, Serv Gastrenterol, Porto, Portugal
Hernández-Guerra, M:
Hosp Univ Canarias, Santa Cruz De Tenerife, Spain
Olivas, I:
Hosp Clin Barcelona, Barcelona, Spain
Rodríguez-Taje, S:
Hosp Clin Barcelona, Barcelona, Spain
Londoño, M:
Hosp Clin Barcelona, Barcelona, Spain
Sousa, JM:
Hosp Univ Virgen Rocio, Seville, Spain
Ampuero, J:
Hosp Univ Virgen Rocio, Seville, Spain
Inst Biomed Sevilla IBiS, Seville, Spain
Romero-González, E:
Univ Valencia, Hosp Clin Univ Valencia, Valencia, Spain
González-Padilla, S:
Univ Valencia, Hosp Clin Univ Valencia, Valencia, Spain
Escudero-García, D:
Univ Valencia, Hosp Clin Univ Valencia, Valencia, Spain
Carvalho, A:
Ctr Hosp & Univ Coimbra, Coimbra, Portugal
Santos, A:
Ctr Hosp & Univ Coimbra, Coimbra, Portugal
Gutiérrez, ML:
Hosp Univ Fdn Alcorcon, Madrid, Spain
Univ Rey Juan Carlos, Madrid, Spain
Pérez-Fernández, E:
Hosp Univ Fdn Alcorcon, Madrid, Spain
Univ Rey Juan Carlos, Madrid, Spain
Aburruza, L:
Hosp Univ Donostia, Donostia San Sebastian, Spain
Uriz, J:
Complejo Hosp Navarra, Pamplona, Spain
Gomes, D:
CHU Coimbra, Dept Gastrenterol, Coimbra, Portugal
Santos, L:
CHU Coimbra, Dept Gastrenterol, Coimbra, Portugal
Martínez-González, J:
Hosp Univ Ramon & Cajal, Madrid, Spain
Albillos, A:
Hosp Univ Ramon & Cajal, Madrid, Spain
Ramon & Cajal Inst Hlth Res, Madrid, Spain
Univ Alcala de Henares, Alcala De Henares, Spain
Fernández-Rodríguez, CM:
Univ Rey Juan Carlos, Hosp Univ Fdn Alcorcon, Madrid, Spain
Hosp Univ Fdn Alcorcon, Madrid, Spain
Univ Rey Juan Carlos, Madrid, Spain
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