Vanishing bile duct syndrome (VBDS) is a rare cholangiopathy that may follow drug-induced liver injury and can progress to liver failure. No established pharmacological treatment is currently available. We present a case of a 65-year-old man diagnosed with histologically confirmed and progressive symptomatic VBDS, probably sustained by drug-induced liver injury, who was successfully treated with a 4-week course of off-label ileal bile acid transporter inhibitor treatment of the management of pruritus. To our knowledge, this is among the first reports of successful ileal bile acid transporter inhibitor use in VBDS. This case highlights the potential role of bile acid modulation in managing severe symptomatic VBDS and underscores the importance of a proactive, multidisciplinary approach to optimize outcomes in complex cholangiopathies.

Primary biliary cholangitis (PBC) is a systemic disease in which musculoskeletal complications may impair function despite satisfactory biochemical control. This narrative review integrates PBC-specific evidence on sarcopenia, osteoporosis and osteosarcopenia with mechanistic studies of cholestasis, muscle-bone interactions and peroxisome proliferator-activated receptor (PPAR) signalling. Muscle depletion occurs before cirrhosis, but heterogeneous definitions and retrospective designs limit prevalence estimates and prognostic interpretation. Population-based studies link PBC to fractures and increased post-fracture mortality, whereas dedicated osteosarcopenia studies remain limited. Nutritional compromise, inflammation, endocrine abnormalities, symptom-related inactivity and advanced liver disease converge on the muscle-bone unit. General densitometric and fracture-risk frameworks remain necessary because PBC-specific integrated thresholds are unvalidated. PPAR agonists improve biochemical activity and, for some agents, symptoms, but their musculoskeletal effects must be considered separately by receptor isoform. Experimental oxidative adaptation and exploratory bone-density findings do not establish sarcopenia reversal or fracture protection; fracture signals and potential muscle toxicity require independent assessment. Ileal bile acid transporter inhibition offers pruritus relief but also requires attention to fat-soluble vitamins and skeletal safety. Integrated evaluation of symptoms, strength, body composition, physical performance and fracture risk may identify actionable limitations. Prospective cohorts and controlled interventions with predefined musculoskeletal endpoints are needed to establish clinically meaningful benefit.