Longitudinal outcomes of obeticholic acid therapy in ursodiol-nonresponsive primary biliary cholangitis: Stratifying the impact of add-on fibrates in real-world practice
Gómez, E. ; Montero, J. L. ; Molina, E. ; García-Buey, L. ; Casado, M. ; Fuentes, J. (Universidad de Zaragoza) ; Simón, M. A. (Universidad de Zaragoza) ; Díaz-González, A. ; Jorquera, F. ; Morillas, R. M. ; Presa, J. ; Berenguer, M. ; Conde, M. I. ; Olveira, A. ; Macedo, G. ; Garrido, I. ; Hernández-Guerra, M. ; Olivas, I. ; Rodríguez-Tajes, S. ; Londoño, M. ; Sousa, J. M. ; Ampuero, J. ; Romero-González, E. ; González-Padilla, Sh. ; Escudero-García, D. ; Carvalho, A. ; Santos, A. ; Gutiérrez, M. L. ; Pérez-Fernández, E. ; Aburruza, L. ; Uriz, J. ; Gomes, D. ; Santos, L. ; Martínez-González, J. ; Albillos, A. ; Fernández-Rodríguez, C. M.
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.
Idioma: Inglés
DOI: 10.1111/apt.18004
Año: 2024
Publicado en: ALIMENTARY PHARMACOLOGY & THERAPEUTICS 59, 12 (2024), 1604-1615
ISSN: 0269-2813
Tipo y forma: Article (Published version)
Área (Departamento): Area Medicina (Dpto. Medicina, Psiqu. y Derm.)
You must give appropriate credit, provide a link to the license, and indicate if changes were made. You may do so in any reasonable manner, but not in any way that suggests the licensor endorses you or your use. You may not use the material for commercial purposes. If you remix, transform, or build upon the material, you may not distribute the modified material.
Exportado de SIDERAL (2024-07-05-12:56:07)
Visitas y descargas
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.
Idioma: Inglés
DOI: 10.1111/apt.18004
Año: 2024
Publicado en: ALIMENTARY PHARMACOLOGY & THERAPEUTICS 59, 12 (2024), 1604-1615
ISSN: 0269-2813
Tipo y forma: Article (Published version)
Área (Departamento): Area Medicina (Dpto. Medicina, Psiqu. y Derm.)
You must give appropriate credit, provide a link to the license, and indicate if changes were made. You may do so in any reasonable manner, but not in any way that suggests the licensor endorses you or your use. You may not use the material for commercial purposes. If you remix, transform, or build upon the material, you may not distribute the modified material. Exportado de SIDERAL (2024-07-05-12:56:07)
Permalink:
Visitas y descargas
Este artículo se encuentra en las siguientes colecciones:
Articles
Record created 2024-05-22, last modified 2024-07-05