Liver stiffness measurement by vibration-controlled transient elastography improves outcome prediction in primary biliary cholangitis
Open accessAbstract
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.
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A large multicentre retrospective study involving 3985 patients showed that LSM assessed by vibration-controlled transient elastography provided a better prognostic ability than biochemical response.
“A large multicentre retrospective study involving 3985 patients showed that LSM assessed by vibration-controlled transient elastography provided a better prognostic ability than biochemical response.124”
From Primary biliary cholangitis. (2024) · cites this paper as [124]
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Self-citationPrimary cilium‐mediated signaling cascade suppresses age‐related biliary fibrosis
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Pharmacologic Treatment of Portal Hypertension
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Optimizing treatment of primary biliary cholangitis: Is good, good enough?
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Self-citationThe unhealthy lifestyle in primary biliary cholangitis: An enemy to fight
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Self-citationCurrent and future opportunities for the management of primary biliary cholangitis
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Primary biliary cholangitis: personalized medicine for optimal therapeutic opportunities
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AI in the Prediction of Hepatic Fibrosis Progression Using Non-Coding RNAs
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Self-citationPrognostic modeling in biliary diseases
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Mechanical mechanics-reclaiming a new battlefield for chronic liver disease
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Self-citationUltrasound imaging findings in primary biliary cholangitis
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Optimizing Care in Primary Biliary Cholangitis: Current Treatments and the Second-Line Decision
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Self-citation
- Authors
- Corpechot, C., Carrat, F., Gaouar, F., Chau, F., Hirschfield, G., Gulamhusein, A., Montano-Loza, A. J., Lytvyak, E., Schramm, C., Pares, A., Olivas, I., Eaton, J. E., Osman, K. T., Dalekos, G., Gatselis, N., Nevens, F., Cazzagon, N., Zago, A., Russo, F. P., 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, A. E., Deibel, A., Dumortier, J., Bruns, T., Große, K., Pageaux, G.-P., Wetten, A., Dyson, J., Jones, D., Chazouillères, O., Hansen, B., & de Lédinghen, V.
- Affiliations
- Inserm (FR)
- Sorbonne Université (FR)
- University Health Network (CA)
- University of Alberta (CA)
- Universität Hamburg (DE)
- Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (ES)
- Mayo Clinic (US)
- University Hospital of Larissa (GR)
- Universitair Ziekenhuis Leuven (BE)
- University of Padua (IT)
- University Hospitals Birmingham NHS Foundation Trust (GB)
- The Royal Free Hospital (GB)
- University of Palermo (IT)
- Centre Hospitalier Universitaire de Bordeaux (FR)
- Beth Israel Deaconess Medical Center (US)
- Università degli Studi del Piemonte Orientale “Amedeo Avogadro” (IT)
- University of Milano-Bicocca (IT)
- Tel Aviv Sourasky Medical Center (IL)
- University Hospital Zurich (CH)
- Hospices Civils de Lyon (FR)
- Universitätsklinikum Aachen (DE)
- Centre Hospitalier Universitaire de Montpellier (FR)
- Newcastle upon Tyne Hospital (GB)
- Corresponding author
- Corpechot, Christophe
- ORCID
- Corpechot, Christophe
- Carrat, Fabrice
- Hirschfield, Gideon
- Gulamhusein, Aliya
- Lytvyak, Ellina
- Schramm, Christoph
- Pares, Albert
- Olivas, Ignasi
- Eaton, John E.
- Osman, Karim T.
- Dalekos, George
- Gatselis, Nikolaos
- Nevens, Frederik
- Cazzagon, Nora
- Zago, Alessandra
- Russo, Francesco Paolo
- Abbas, Nadir
- Trivedi, Palak
- Thorburn, Douglas
- Saffioti, Francesca
- Roccarina, Davide
- Delamarre, Adèle
- Medina-Morales, Esli
- Bonder, Alan
- Patwardhan, Vilas
- Rigamonti, Cristina
- Carbone, Marco
- Invernizzi, Pietro
- Cristoferi, Laura
- van der Meer, Adriaan
- Zigmond, Ehud
- Yehezkel, Eyal
- Kremer, Andreas E.
- Deibel, Ansgar
- Dumortier, Jérôme
- Bruns, Tony
- Große, Karsten
- Pageaux, Georges-Philippe
- Wetten, Aaron
- Dyson, Jessica
- Chazouillères, Olivier
- Venue
- Journal of Hepatology
- Publisher
- Elsevier BV
- Published
- 2022-12
- Volume
- 77
- Issue
- 6
- Pages
- 1545-1553
- ISSN
- 0168-8278 (ISSN-L)
- OpenAlex
- W4283645165
- Cited by
- 128
- References
- 40
- Citations by year
- 2026: 30 · 2025: 40 · 2024: 27 · 2023: 25 · 2022: 6
- FWCI
- 14.61
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Journal of Hepatology (acceptedVersion)
- PubMed (publishedVersion)
- EUR Research Repository (Erasmus University Rotterdam) (publishedVersion, cc-by-nc-nd)
- Lirias (KU Leuven) (submittedVersion, other-oa)
- EUR Research Repository (Erasmus University Rotterdam) (submittedVersion, cc-by-nc-nd)
- HAL (submittedVersion, other-oa)
- BOA (University of Milano-Bicocca) (submittedVersion)
- UCL Discovery (University College London) (submittedVersion)
- Nova Science Publishers (Nova Science Publishers, Inc.) (submittedVersion)
- RWTH Publications (RWTH Aachen) (submittedVersion)
- Padua Research Archive (University of Padova) (submittedVersion)
- Zurich Open Repository and Archive (University of Zurich) (submittedVersion)
- Newcastle University ePrints (Newcastle Univesity)
- Funders
- Falk Foundation
- GlaxoSmithKline
- Inventiva Pharma
- National Institute for Health and Care Research (NIHR200243, NF-SI-0513-10091)
- Swedish Orphan Biovitrum
- Grifols
- Medical Research Council (MR/L001489/1)
- Topics
- Health Sciences › Medicine › Hepatology › Liver Diseases and Immunity
- Health Sciences › Medicine › Epidemiology › Liver Disease Diagnosis and Treatment
- Health Sciences › Medicine › Surgery › Pediatric Hepatobiliary Diseases and Treatments
- Keywords
- primary biliary cirrhosis, liver stiffness measurement, vibration-controlled transient elastography, prognosis, liver complications, liver transplantation, mortality, liver fibrosis, surrogate endpoints, risk stratification, cut-off values, prognostic validation, fibrosis score, autoimmune liver disease, time-dependent Cox regression, concordance index
- MeSH terms
- Follow-Up Studies, Humans, Liver, Liver Cirrhosis, Liver Cirrhosis, Biliary, Prognosis, Retrospective Studies, Vibration, Cohort Studies, Elasticity Imaging Techniques
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