FRI-057-A review of primary biliary cholangitis practice in Wales: Time for specialist care

Haboubi, Hasan; Shenbagaraj, Lavanya; Abdul-Sattar, Asma; Harborne, Philip; Philips, A.; Appanna, Gautham; Yahya, Imran; Navaratnam, Janardhan; Edwards, Kate; Alrubaiy, Laith; Newbould, Rachel; Vincent, Rebecca; Mannem, Seetharam; Gardezi, Syed Anjum Ali; Aslam, Usama; Jennings, Victoria; Samuel, D; Pembroke, Tom; Ch’ng, Chin Lye; Srivastava, Brijesh; Yousuf, Fidan; Yeoman, Andrew D.

doi:10.1016/s0618-8278(19)30807-2

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“…The strong association between UDCA therapy and prolonged liver transplantation (LT)-free survival was recently substantiated in both a large American cohort and our own international cohort, with a dose–response relationship highlighting the importance of the 13–15 mg/kg dose recommendation 8. Still, even in recent Western cohorts, as much as 30% of patients remained untreated and suboptimal UDCA dosages were frequently used 9 10. More awareness of and attention for the clinical efficacy of UDCA are thus needed in order to optimise the medical management and clinical outcome of the population with PBC.…”

Section: Introductionmentioning

confidence: 97%

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Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis

Harms

Veer

Lammers

et al. 2019

Gut

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ObjectiveThe clinical benefit of ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC) has never been reported in absolute measures. The aim of this study was to assess the number needed to treat (NNT) with UDCA to prevent liver transplantation (LT) or death among patients with PBC.MethodsThe NNT was calculated based on the untreated LT-free survival and HR of UDCA with respect to LT or death as derived from inverse probability of treatment weighting-adjusted Cox proportional hazard analyses within the Global PBC Study Group database.ResultsWe included 3902 patients with a median follow-up of 7.8 (4.1–12.1) years. The overall HR of UDCA was 0.46 (95% CI 0.40 to 0.52) and the 5-year LT-free survival without UDCA was 81% (95% CI 79 to 82). The NNT to prevent one LT or death within 5 years (NNT5y) was 11 (95% CI 9 to 13). Although the HR of UDCA was similar for patients with and without cirrhosis (0.33 vs 0.31), the NNT5y was 4 (95% CI 3 to 5) and 20 (95% CI 14 to 34), respectively. Among patients with low alkaline phosphatase (ALP) (≤2× the upper limit of normal (ULN)), intermediate ALP (2–4× ULN) and high ALP (>4× ULN), the NNT5y to prevent one LT or death was 26 (95% CI 15 to 70), 11 (95% CI 8 to 17) and 5 (95% CI 4 to 8), respectively.ConclusionThe absolute clinical efficacy of UDCA with respect to LT or death varied with baseline prognostic characteristics, but was high throughout. These findings strongly emphasise the incentive to promptly initiate UDCA treatment in all patients with PBC and may improve patient compliance.

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Section: Introductionmentioning

confidence: 97%

“… 8 Still, even in recent Western cohorts, as much as 30% of patients remained untreated and suboptimal UDCA dosages were frequently used. 9 10 More awareness of and attention for the clinical efficacy of UDCA are thus needed in order to optimise the medical management and clinical outcome of the population with PBC.…”

Section: Introductionmentioning

confidence: 99%