Liver and Bile

J Hepatol. 2022;77(6):1545–53

Corpechot C, Carrat F, Gaouar F, Chau F, Hirschfield G, Gulamhusein A, Montano-Loza AJ, Lytvyak E, Schramm C, Pares A, Olivas I, Eaton JE, Osman KT, Dalekos G, Gatselis N, Nevens F, Cazzagon N, Zago A, Russo FP, Abbas N, Trivedi P, Thorburn D, Saffioti F, Barkai L, Roccarina D, Calvaruso V, Fichera A, Delamarre A, Medina-Morales E, Bonder A, Patwardhan V, Rigamonti C, Carbone M, Invernizzi P, Cristoferi L, van der Meer A, de Veer R, Zigmond E, Yehezkel E, Kremer AE, Deibel A, Dumortier J, Bruns T, Große K, Pageaux GP, Wetten A, Dyson J, Jones D, Chazouillères O, Hansen B, de Lédinghen V; Global & ERN Rare-Liver PBC Study Groups

Liver stiffness measurement by vibration-controlled transient elastography improves outcome prediction in primary biliary cholangitis

Background and aims: Liver stiffness measurement (LSM) by vibration-controlled transient elastography (VCTE) has been shown to predict outcomes of patients with primary biliary cholangitis (PBC) in small-size studies. The aim of this study was to validate the prognostic value of LSM in a large cohort study.
Methods: The authors performed an international, multicenter, retrospective follow-up study of 3985 patients with PBC seen at 23 centers in 12 countries. Eligibility criteria included at least 1 reliable LSM by VCTE and a follow-up ≥ 1 year. Independent derivation (n = 2740) and validation (n = 568) cohorts were built. The primary end point was time to poor clinical outcomes defined as liver-related complications, liver transplantation, or death. Hazard ratios (HRs) with 95%-confidence intervals (CIs) were determined using a time-dependent multivariable Cox regression analysis.
Results: LSM was independently associated with poor clinical outcomes in the derivation (5324 LSMs, mean follow-up 5.0 ± 3.1 years) and validation (1470 LSMs, mean follow-up 5.0 ± 2.8 years) cohorts: adjusted HRs (95% CI) per additional kPa were 1.040 (1.026–1.054) and 1.042 (1.029–1.056), respectively (p < 0.0001 for both). Adjusted C-statistics (95% CI) at baseline were 0.83 (0.79–0.87) and 0.92 (0.89–0.95), respectively. Between 5 and 30 kPa, the log-HR increased as a monotonic function of LSM. The predictive value of LSM was stable in time. LSM improved the prognostic ability of biochemical response criteria, fibrosis scores, and prognostic scores. The 8-kPa and 15-kPa cut-offs optimally separated low-, medium-, and high-risk groups. 40% of patients were at medium to high risk according to LSM.

Conclusions: Liver stiffness measurement by vibration-controlled transient elastography is a major, independent, validated predictor of primary biliary cholangitis (PBC) outcome. Its value as a surrogate end point for clinical benefit in PBC should be considered.

DOI: DOI: 10.1016/j.jhep.2022.06.017