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

Research output: Contribution to journalArticlepeer-review


  • Maren H Harms
  • Rozanne C de Veer
  • Willem J Lammers
  • Christophe Corpechot
  • Douglas Thorburn
  • Harry L A Janssen
  • Keith D Lindor
  • Albert Pares
  • Annarosa Floreani
  • Marlyn J Mayo
  • Pietro Invernizzi
  • Pier Maria Battezzati
  • Frederik Nevens
  • Cyriel Y Ponsioen
  • Andrew L Mason
  • Kris V Kowdley
  • Bettina E Hansen
  • Henk R van Buuren
  • Adriaan J van der Meer

Colleges, School and Institutes

External organisations

  • Erasmus University Medical Center
  • Hôpital Saint-Antoine, Service d'Hépatologie, INSERM, UMR_S 938, CDR Saint-Antoine, and Sorbonne Universités, UPMC Univ Paris 06, Paris, France.
  • The Sheila Sherlock Liver Centre and UCL Institute for Liver and Digestive Health
  • Toronto Centre for Liver Disease
  • Department of Gastroenterology and Hepatology, Erasmus MC University Medical Centre
  • University of Barcelona
  • Oxford Colorectal Surgery Department, Nuffield Department of Surgery, Churchill Hospital, Oxford, Oxfordshire, UK.
  • Program for Autoimmune Liver Diseases, International Center for Digestive Health, Department of Medicine and Surgery, University of Milan-Bicocca, Milan, Italy.
  • University of Milan
  • Amsterdam University Medical Centres
  • University of Alberta
  • Liver Care Network and Organ Care Research


OBJECTIVE: 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.

METHODS: 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.

RESULTS: 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 (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.

CONCLUSION: 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.

Bibliographic note

© Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY. Published by BMJ.


Original languageEnglish
Early online date16 Dec 2019
Publication statusE-pub ahead of print - 16 Dec 2019