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

The 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. The 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. We 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 (NNT The 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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Competing interests: MHH reports a speaker fee from Zambon Nederland. WL reports consulting services for Intercept Pharmaceuticals. CC is a consultant for Intercept Pharmaceuticals France. DT reports consulting activities for Intercept Pharmaceuticals. HLAJ reports grants from and consulting work for AbbVie Pharmaceuticals, Bristol-Myers Squibb, Gilead Sciences, Innogenetics, Merck, Novartis, Roche, Intercept Pharmaceuticals and Janssen. KL reports that he is an unpaid advisor for Intercept Pharmaceuticals and Shire. PT receives institutional salary support from the NIHR Birmingham Liver Biomedical Research Centre. He received research grant funding from the Wellcome Trust, Guts UK, PSC Support and Intercept Pharmaceuticals. He also received advisory and consultancy fees from Intercept and Dr Falk Pharma, and speaker fees from Intercept, Dr Falk Pharma, Zambon and Perspectum Diagnostics. GH reports advisory services for Intercept Pharmaceuticals, Novartis and GlaxoSmithKline Pharmaceuticals. AP reports consulting services for Intercept Pharmaceuticals and Novartis Pharma. AF reports consulting activities for Intercept Pharmaceuticals. PI reports personal fees from Intercept and non-financial support from Bruschettini and Menarini Diagnostics. CYP has received grant support form Takeda, speaker’s fees from AbbVie, Takeda and Dr Falk Pharma, and served as a consultant for Takeda and Pliant. AM reports advisory services for Intercept Pharmaceuticals, AbbVie and Novartis, and research funding resources from the Canadian Institutes of Health Research, Canadian Liver Foundation, American Kennel Club, Intercept Pharmaceuticals, AbbVie and Gilead Sciences. KK reports personal fees from Gilead Sciences, Intercept Pharmaceuticals and Novartis, and grants from Gilead Sciences and Intercept Pharmaceuticals. BEH reports grants from Intercept Pharmaceuticals and Zambon Nederland, and consulting work for Intercept Pharmaceuticals and Novartis. HvB is a consultant for Intercept Pharma Benelux and received unrestricted research grants from Intercept Pharmaceuticals and from Zambon Nederland. AJvdM reports speaker's fees from MSD, Gilead Sciences, AbbVie Pharmaceuticals and Zambon Nederland, received an unrestricted grant from Gilead Sciences, and reports travel expenses covered by Dr Falk Pharma.