Longitudinal outcomes of obeticholic acid therapy in ursodiol-nonresponsive primary biliary cholangitis

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 effectivenes...

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Autores: Gómez, Elena, Montero, José Luís, Molina Pérez, Esther|||0000-0002-6951-3600, García-Buey, Luisa, Casado Martín, Marta D., Fuentes, Javier, Simón Marco, Miguel Ángel, Díaz-González, Álvaro|||0000-0003-3610-2263, Jorquera, Francisco|||0000-0002-1799-2881, Morillas, Rosa María A., Presa, José, Berenguer, Marina|||0000-0001-9246-4264, Conde, Manuel I., Olveira, Antonio|||0000-0002-2991-4688, Macedo, Guilherme, Garrido, Isabel|||0000-0002-7801-466X, Hernandez-Guerra, Manuel|||0000-0002-3478-9981, Olivas, Ignasi, Rodríguez-Tajes, Sergio|||0000-0002-3189-7557, Londoño, Maria Carlota|||0000-0002-6533-1586, Sousa, José Manuel, Ampuero, Javier|||0000-0002-8332-2122, Romero-González, E., González-Padilla, Sh, Escudero-García, Desamparados, Carvalho, Armando|||0000-0003-2455-8781, Santos, Arsénio|||0000-0001-5091-5223, Gutiérrez-García, María Luisa, Perez-Fernandez, Elia|||0000-0003-2726-8683, Aburruza, Leire, Úriz, Javier, Gomes, Dário, Santos, Luis, Martínez-González, Javier, Albillos, Agustín|||0000-0001-9131-2592, Fernández-Rodríguez, Conrado Manuel
Tipo de recurso: artículo
Fecha de publicación:2024
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:301946
Acceso en línea:https://ddd.uab.cat/record/301946
https://dx.doi.org/urn:doi:10.1111/apt.18004
Access Level:acceso abierto
Palabra clave:Adult
Aged
Alkaline Phosphatase
Bilirubin
Chenodeoxycholic Acid
Cholagogues and Choleretics
Drug Therapy, Combination
Female
Fibric Acids
Humans
Liver Cirrhosis, Biliary
Longitudinal Studies
Male
Middle Aged
Spain
Treatment Outcome
Ursodeoxycholic Acid
Descripción
Sumario: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.

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