TY - JOUR
T1 - Optimizing therapy in primary biliary cholangitis
T2 - Alkaline phosphatase at six months identifies one-year non-responders and predicts survival
AU - the GLOBAL PBC Study Group
AU - Murillo Perez, C. Fiorella
AU - Ioannou, Stephanie
AU - Hassanally, Iman
AU - Trivedi, Palak J.
AU - Corpechot, Christophe
AU - van der Meer, Adriaan J.
AU - Lammers, Willem J.
AU - Battezzati, Pier Maria
AU - Lindor, Keith D.
AU - Nevens, Frederik
AU - Kowdley, Kris V.
AU - Bruns, Tony
AU - Cazzagon, Nora
AU - Floreani, Annarosa
AU - Mason, Andrew L.
AU - Gulamhusein, Aliya
AU - Ponsioen, Cyriel Y.
AU - Carbone, Marco
AU - Lleo, Ana
AU - Mayo, Marlyn J.
AU - Dalekos, George N.
AU - Gatselis, Nikolaos K.
AU - Thorburn, Douglas
AU - Verhelst, Xavier
AU - Parés, Albert
AU - Londoño, Maria Carlota
AU - Janssen, Harry L.A.
AU - Invernizzi, Pietro
AU - Vuppalanchi, Raj
AU - Hirschfield, Gideon M.
AU - Hansen, Bettina E.
AU - Levy, Cynthia
N1 - Publisher Copyright:
© 2023 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
PY - 2023/7
Y1 - 2023/7
N2 - Background and Aims: Patients with primary biliary cholangitis (PBC) and insufficient response to ursodeoxycholic acid (UDCA), currently assessed after 1 year, are candidates for second-line therapy. The aims of this study are to assess biochemical response pattern and determine the utility of alkaline phosphatase (ALP) at six months as a predictor of insufficient response. Methods: UDCA-treated patients in the GLOBAL PBC database with available liver biochemistries at one year were included. POISE criteria were used to assess response to treatment, defined as ALP <1.67 × upper limit of normal (ULN) and normal total bilirubin at one year. Various thresholds of ALP at six months were evaluated to predict insufficient response based on negative predictive value (NPV) and that with nearest to 90% NPV was selected. Results: For the study, 1362 patients were included, 1232 (90.5%) female, mean age of 54 years. The POISE criteria were met by 56.4% (n = 768) of patients at one year. The median ALP (IQR) of those who met POISE criteria compared to those who did not was 1.05 × ULN (0.82–1.33) vs. 2.37 × ULN (1.72–3.69) at six months (p <.001). Of 235 patients with serum ALP >1.9 × ULN at six months, 89% did not achieve POISE criteria (NPV) after one year of UDCA. Of those with insufficient response by POISE criteria at one year, 210 (67%) had an ALP >1.9 × ULN at six months and thus would have been identified early. Conclusions: We can identify patients for second-line therapy at six months using an ALP threshold of 1.9 × ULN, given that approximately 90% of these patients are non-responders according to POISE criteria.
AB - Background and Aims: Patients with primary biliary cholangitis (PBC) and insufficient response to ursodeoxycholic acid (UDCA), currently assessed after 1 year, are candidates for second-line therapy. The aims of this study are to assess biochemical response pattern and determine the utility of alkaline phosphatase (ALP) at six months as a predictor of insufficient response. Methods: UDCA-treated patients in the GLOBAL PBC database with available liver biochemistries at one year were included. POISE criteria were used to assess response to treatment, defined as ALP <1.67 × upper limit of normal (ULN) and normal total bilirubin at one year. Various thresholds of ALP at six months were evaluated to predict insufficient response based on negative predictive value (NPV) and that with nearest to 90% NPV was selected. Results: For the study, 1362 patients were included, 1232 (90.5%) female, mean age of 54 years. The POISE criteria were met by 56.4% (n = 768) of patients at one year. The median ALP (IQR) of those who met POISE criteria compared to those who did not was 1.05 × ULN (0.82–1.33) vs. 2.37 × ULN (1.72–3.69) at six months (p <.001). Of 235 patients with serum ALP >1.9 × ULN at six months, 89% did not achieve POISE criteria (NPV) after one year of UDCA. Of those with insufficient response by POISE criteria at one year, 210 (67%) had an ALP >1.9 × ULN at six months and thus would have been identified early. Conclusions: We can identify patients for second-line therapy at six months using an ALP threshold of 1.9 × ULN, given that approximately 90% of these patients are non-responders according to POISE criteria.
KW - early identification
KW - monitoring
KW - primary biliary cholangitis
KW - prognosis
KW - treatment
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U2 - 10.1111/liv.15592
DO - 10.1111/liv.15592
M3 - Article
C2 - 37157905
AN - SCOPUS:85158967611
SN - 1478-3223
VL - 43
SP - 1497
EP - 1506
JO - Liver International
JF - Liver International
IS - 7
ER -