Liver transplant‐free survival according to alkaline phosphatase and <scp>GLOBE</scp> score in patients with primary biliary cholangitis treated with ursodeoxycholic acid
Rozanne C. de Veer, Maren H. Harms, Christophe Corpechot, Douglas Thorburn, Pietro Invernizzi, Harry L.A. Janssen, Pier Maria Battezzati, Frederik Nevens, Keith D. Lindor, Annarosa Floreani, Cyriel Y. Ponsioen, Marlyn J. Mayo, Albert Parés, Andrew L. Mason, Kris V. Kowdley, Palak Trivedi, Gideon M. Hirschfield, Tony Bruns, George Ν. Dalekos, Nikolaos Gatselis, Xavier Verhelst, Willem J. Lammers, Bettina E. Hansen, Henk R. van Buuren, Adriaan J. van der Meer, the Global PBC Study Group
Abstract
BACKGROUND: After 1 year of ursodeoxycholic acid (UDCA), patients with primary biliary cholangitis (PBC) may have a normal GLOBE score despite high alkaline phosphatase (ALP) levels. AIM: To assess the association between ALP and liver transplantation (LT)-free survival according to the GLOBE score METHODS: Among patients with a normal or elevated GLOBE score in the Global PBC cohort, the association between ALP after 1 year of UDCA and the risk of LT/death was assessed. The LT-free survival was compared with that of a matched general population. RESULTS: After 1 year of UDCA, ALP was associated with the risk of LT/death (aHR 1.31, 95% CI 1.003-1.72, p = 0.048) among 2729 patients with a normal GLOBE score. The 10-year LT-free survival among these patients with an ALP >2.0 × ULN was 94.0% (95% CI 90.1-97.9) for those <50 years, and 82.6% (95% CI 76.5-88.7) for those ≥50 years, which was significantly lower (p = 0.040) and similar (p = 0.736) to that of the matched population, respectively. The 10-year LT-free survival in patients ≥50 years with normal GLOBE score and normal ALP (90.8%, 95% CI 87.7-93.9) was significantly higher (p = 0.022) than the matched population. Among 1045 patients with an elevated GLOBE score, ALP was associated with LT/death only in those <50 years (aHR 1.38, 95% CI 1.06-1.81, p = 0.016). CONCLUSION: The LT-free survival of patients with PBC with a normal GLOBE score is optimal in case of normal ALP levels, also in relation to the general population. Despite their generally favourable prognosis, an elevated ALP level may still indicate a need for add-on therapy.