Long‐term clinical impact and cost‐effectiveness of obeticholic acid for the treatment of primary biliary cholangitis

Samur, Sumeyye; Klebanoff, Matthew J.; Banken, Reiner; Pratt, Daniel S.; Chapman, Rick; Ollendorf, Daniel A.; Loos, Anne M.; Corey, Kathleen E.; Hur, Chin; Chhatwal, Jagpreet

doi:10.1002/hep.28932

Cited by 75 publications

(42 citation statements)

References 31 publications

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“…Thus, OCA may be prescribed lifelong along with UDCA. Based on the high cost of OCA treatment (US $69,350 per year), the unlimited prescription of OCA would pose a substantial economic burden on patients and societies …”

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confidence: 99%

Bezafibrate Improves GLOBE and UK‐PBC Scores and Long‐Term Outcomes in Patients With Primary Biliary Cholangitis

et al. 2019

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In Japan, bezafibrate (BF) is a second‐line agent for primary biliary cholangitis (PBC) that is refractory to ursodeoxycholic acid (UDCA) treatment. From a retrospective cohort (n = 873) from the Japan PBC Study Group, we enrolled 118 patients who had received UDCA monotherapy for at least 1 year followed by combination therapy with UDCA+BF for at least 1 year. GLOBE and UK‐PBC scores after UDCA monotherapy (i.e., immediately before UDCA+BF combination therapy) were compared with those after 1 year of UDCA+BF combination therapy. The real outcomes of enrolled patients estimated by Kaplan–Meier analysis were compared with the predicted outcomes calculated using GLOBE and UK‐PBC scores. In addition, the hazard ratio of BF treatment was calculated using propensity score analysis. The mean GLOBE score before the combination therapy was 0.504 ± 0.080, which improved significantly to 0.115 ± 0.085 (P < 0.0001) after 1 year of combination therapy. The real liver transplant‐free survival of enrolled patients was significantly better than that predicted by GLOBE score before introducing BF. Combination therapy did not significantly improve the real rates of liver transplantation or liver‐related death compared with those predicted by UK‐PBC risk score before introducing BF, but the predicted risk was significantly reduced by the addition of BF (P < 0.0001). Cox regression analysis with inverse probability of treatment weighting showed that the addition of BF significantly reduced the hazard of liver transplant or liver‐related death in patients who, after 1 year of UDCA monotherapy, had normal serum bilirubin (adjusted hazard ratio 0.09, 95% confidence interval 0.01‐0.60, P = 0.013). Conclusion: Addition of BF to UDCA monotherapy improves not only GLOBE and UK‐PBC scores but also the long‐term prognosis of PBC patients, especially those with early‐stage PBC.

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confidence: 99%

Bezafibrate Improves GLOBE and UK‐PBC Scores and Long‐Term Outcomes in Patients With Primary Biliary Cholangitis

et al. 2019

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“…The addition of OCA therapy to UDCA therapy in PBC patients would decrease the 15-year cumulative incidence of liver-related deaths from 27% to 11%, the cumulative incidence of decompensated cirrhosis from 5% to 2%, the cumulative incidence of hepatocellular carcinoma (HCC) from 4% to 2%, and the cumulative incidence of liver transplant from 0.6% to 0.2%. OCA treatment would also increase the 15-year transplant-free survival rate from 60% to 72% [33]. These data suggest that the use of OCA is expected to improve the long-term outcome in PBC patients.…”

Section: Obeticholic Acidmentioning

confidence: 70%

“…Estimation of OCA long-term clinical impact and costeffectiveness has been assessed with a microsimulation model in an adult population with inadequate response to UDCA with reference to the titration arm of POISE [33]. This model was validated with 15-year transplant-free survival data from the Global PBC Study.…”

Section: Obeticholic Acidmentioning

confidence: 99%

Geoepidemiology and changing mortality in primary biliary cholangitis

et al. 2017

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“…Although use of OCA leads to signifi cant improvement in serum alkaline phosphatase (ALP) in a subset of patients, long-term benefi ts of OCA have only been demonstrated through mathematical modeling ( 2 ). Ongoing studies are awaited to confi rm such benefi t (NCT02308111).…”

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confidence: 99%

“…is not yet widely available, requires caution, and dose adjustment in patients with advanced liver disease, and its costs are considered excessive ( 2 ). In this issue of AJG , Reig et al ( 3 ) describe the benefi ts of long-term use of bezafi brate for patients with PBC and incomplete response to UDCA.…”

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confidence: 99%