6533b85dfe1ef96bd12be963

RESEARCH PRODUCT

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

Christophe CorpechotFabrice CarratFarid GaouarFrederic ChauGideon HirschfieldAliya GulamhuseinAldo J. Montano-lozaEllina LytvyakChristoph SchrammAlbert ParésIgnasi OlivasJohn E. EatonKarim T. OsmanGeorge DalekosNikolaos GatselisFrederik NevensNora CazzagonAlessandra ZagoFrancesco Paolo RussoNadir AbbasPalak TrivediDouglas ThorburnFrancesca SaffiotiLaszlo BarkaiDavide RoccarinaVicenza CalvarusoAnna FicheraAdèle DelamarreEsli Medina-moralesAlan BonderVilas PatwardhanCristina RigamontiMarco CarbonePietro InvernizziLaura CristoferiAdriaan Van Der MeerRozanne De VeerEhud ZigmondEyal YehezkelAndreas E. KremerAnsgar DeibelJérôme DumortierTony BrunsKarsten GroßeGeorges-philippe PageauxAaron WettenJessica DysonDavid JonesOlivier ChazouillèresBettina HansenVictor De Lédinghen

subject

Liver CirrhosisFibroScan; Mortality; PBC; Prognosis; TransplantationTransplantationHepatologyFibroScanPrognosiLiver Cirrhosis BiliaryLiver CirrhosiPrognosisPBCVibrationFollow-Up StudieCohort StudiesLiverElasticity Imaging TechniqueRetrospective StudieHumansElasticity Imaging TechniquesMortalityCohort StudieRetrospective StudiesFollow-Up StudiesHuman

description

BACKGROUND & 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. We aimed to validate the prognostic value of LSM in a large cohort study. METHODS: We performed an international, multicentre, retrospective follow-up study of 3,985 patients with PBC seen at 23 centres in 12 countries. Eligibility criteria included at least 1 reliable LSM by VCTE and a follow-up ≥ 1 year. Independent derivation (n = 2,740) and validation (n = 568) cohorts were built. The primary endpoint was time to poor clinical outcomes defined as liver-related complications, liver transplantation, or death. Hazard ratios (HRs) with CIs were determined using a time-dependent multivariable Cox regression analysis. RESULTS: LSM was independently associated with poor clinical outcomes in the derivation (5,324 LSMs, mean follow-up 5.0 ± 3.1 years) and validation (1,470 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. Forty percent of patients were at medium to high risk according to LSM. CONCLUSIONS: LSM by VCTE is a major, independent, validated predictor of PBC outcome. Its value as a surrogate endpoint for clinical benefit in PBC should be considered. LAY SUMMARY: Primary biliary cholangitis (PBC) is a chronic autoimmune disease, wherein the body's immune system mistakenly attacks the bile ducts. PBC progresses gradually, so surrogate markers (markers that predict clinically relevant outcomes like the need for a transplant or death long before the event occurs) are often needed to expedite the drug development and approval process. Herein, we show that liver stiffness measurement is a strong predictor of clinical outcomes and could be a useful surrogate endpoint in PBC trials. ispartof: JOURNAL OF HEPATOLOGY vol:77 issue:6 pages:1545-1553 ispartof: location:Netherlands status: published

yearjournalcountryeditionlanguage
2022-06-27
10.1016/j.jhep.2022.06.017https://hdl.handle.net/10447/574305