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Observational Study
. 2024 Jun;59(12):1604-1615.
doi: 10.1111/apt.18004. Epub 2024 May 1.

Longitudinal outcomes of obeticholic acid therapy in ursodiol-nonresponsive primary biliary cholangitis: Stratifying the impact of add-on fibrates in real-world practice

E Gómez  1 , J L Montero  2 , E Molina  3 , L García-Buey  4 , M Casado  5 , J Fuentes  6 , M A Simón  7   8 , A Díaz-González  9 , F Jorquera  10 , R M Morillas  11 , J Presa  12 , M Berenguer  13   14 , M I Conde  13 , A Olveira  15 , G Macedo  16 , I Garrido  16 , M Hernández-Guerra  17 , Ignasi Olivas  18 , S Rodríguez-Tajes  18 , M Londoño  18 , J M Sousa  19 , J Ampuero  19   20 , E Romero-González  21 , Sh González-Padilla  21 , D Escudero-García  21 , A Carvalho  22 , A Santos  22 , M L Gutiérrez  23   24 , E Pérez-Fernández  23   24 , L Aburruza  25 , J Uriz  26 , D Gomes  27 , L Santos  27 , J Martínez-González  28 , A Albillos  28   29   30 , C M Fernández-Rodríguez  23   24
Affiliations
Observational Study

Longitudinal outcomes of obeticholic acid therapy in ursodiol-nonresponsive primary biliary cholangitis: Stratifying the impact of add-on fibrates in real-world practice

E Gómez et al. Aliment Pharmacol Ther. 2024 Jun.

Abstract

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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References

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