REAL-WORLD ASSESSMENT OF LIVER STIFFNESS MEASUREMENT AND THRESHOLD DEFINITIONS IN PRIMARY BILIARY CHOLANGITIS: A TARGETED LITERATURE REVIEW
Author(s)
Chong H Kim, MPH, MS, PhD1, Kevin Kallmes, MA, JD2, Ranita Meena Tarchand, BA, MS3, Anuj Mubayi, PhD4.
1Gilead, Foster City, CA, USA, 2Nested Knowledge, St. Paul, MN, USA, 3Nested Knowledge, St Paul, MN, USA, 4EviValue, Tempe, AZ, USA.
1Gilead, Foster City, CA, USA, 2Nested Knowledge, St. Paul, MN, USA, 3Nested Knowledge, St Paul, MN, USA, 4EviValue, Tempe, AZ, USA.
OBJECTIVES: Liver stiffness (LS) is increasingly used as a noninvasive structural biomarker to stage fibrosis and monitor disease progression in primary biliary cholangitis (PBC). However, heterogeneity in LS measurement and threshold definitions may limit comparability across real‑world evidence (RWE) studies and hinder consistent clinical decision‑making. This review evaluated LS modalities, threshold definitions, and complementary biochemical assessments in observational PBC studies to identify standardization gaps relevant to RWE and HEOR.
METHODS: A targeted literature review (2000-2026) identified observational studies reporting LS in adults with PBC. Extracted variables included study design, setting, sample size, LS modality, LS thresholds for fibrosis staging, and use of complementary biochemical assessments (e.g., ALT, AST, bilirubin, APRI, and FIB‑4). Findings were synthesized to evaluate methodological variability and its implications for structural assessment in RWE.
RESULTS: Thirty‑four studies met inclusion criteria, with sample sizes ranging from 16 to 3,078 patients; most enrolled 100-1,000 participants. Sixty‑five percent were single‑center studies. Vibration‑controlled transient elastography (VCTE; FibroScan®) was the predominant modality (23/34). Higher bilirubin levels were frequently reported among patients with advanced fibrosis, adverse outcomes, or inadequate treatment response. Reporting of direct associations between ALP and LS was limited. Only 4 studies incorporated complementary biochemical fibrosis assessments alongside LS. Eight studies reported LS thresholds to distinguish advanced from non‑advanced fibrosis, but definitions varied substantially. Most studies used LS thresholds of 10-12.5 kPa (range: ~8 to >15 kPa).
CONCLUSIONS: LS is an established noninvasive structural biomarker in PBC, yet real‑world LS measurement practices and fibrosis thresholds remain highly variable. This variability may reduce comparability across RWE studies, influence risk stratification, and introduce uncertainty into disease progression models and HTA decision-making. Standardizing LS thresholds and reporting would strengthen real‑world evidence generation and support more consistent clinical and payer decision‑making in PBC.
METHODS: A targeted literature review (2000-2026) identified observational studies reporting LS in adults with PBC. Extracted variables included study design, setting, sample size, LS modality, LS thresholds for fibrosis staging, and use of complementary biochemical assessments (e.g., ALT, AST, bilirubin, APRI, and FIB‑4). Findings were synthesized to evaluate methodological variability and its implications for structural assessment in RWE.
RESULTS: Thirty‑four studies met inclusion criteria, with sample sizes ranging from 16 to 3,078 patients; most enrolled 100-1,000 participants. Sixty‑five percent were single‑center studies. Vibration‑controlled transient elastography (VCTE; FibroScan®) was the predominant modality (23/34). Higher bilirubin levels were frequently reported among patients with advanced fibrosis, adverse outcomes, or inadequate treatment response. Reporting of direct associations between ALP and LS was limited. Only 4 studies incorporated complementary biochemical fibrosis assessments alongside LS. Eight studies reported LS thresholds to distinguish advanced from non‑advanced fibrosis, but definitions varied substantially. Most studies used LS thresholds of 10-12.5 kPa (range: ~8 to >15 kPa).
CONCLUSIONS: LS is an established noninvasive structural biomarker in PBC, yet real‑world LS measurement practices and fibrosis thresholds remain highly variable. This variability may reduce comparability across RWE studies, influence risk stratification, and introduce uncertainty into disease progression models and HTA decision-making. Standardizing LS thresholds and reporting would strengthen real‑world evidence generation and support more consistent clinical and payer decision‑making in PBC.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD46
Topic
Health Technology Assessment, Methodological & Statistical Research, Real World Data & Information Systems
Topic Subcategory
Reproducibility & Replicability
Disease
Gastrointestinal Disorders, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases