Diagnostic outcome
Measurement of 24-hour urinary copper excretion revealed markedly elevated values. Subsequent slit-lamp examination demonstrated brownish-green corneal deposits at the level of Descemet’s membrane, consistent with Kayser–Fleischer rings. Genetic analysis confirmed pathogenic variants affecting copper transport. The constellation of findings established the diagnosis of Wilson disease with combined hepatic and neurological involvement.
The diagnostic framework was consistent with the Leipzig scoring system, integrating clinical features (Kayser–Fleischer rings, neurological signs), biochemical markers (reduced ceruloplasmin, elevated urinary copper), and genetic testing.
Where available, newer biomarkers of copper metabolism (such as exchangeable copper parameters) may further support diagnostic evaluation.
Clinical course and complications
At the time of diagnosis, neurological manifestations progressed rapidly, with worsening tremor, dysarthria, and gait instability. Hepatic involvement also evolved, as evidenced by declining synthetic function:
- INR progressively rising from 1.2 to 1.8
- Serum albumin: reduced
Episodes of haemolytic anaemia became more pronounced, reflecting systemic effects of copper toxicity.
Chelation therapy was initiated promptly following diagnosis. However, neurological recovery remained incomplete, consistent with observations that advanced neurological injury may be only partially reversible. Progressive hepatic dysfunction ultimately required referral for liver transplantation assessment.
Clinical reasoning and discussion
This case illustrates how persistent but mild liver test abnormalities may lead to diagnostic inertia, particularly when imaging findings such as steatosis appear to provide a plausible explanation. In young adults, metabolic-associated steatotic liver disease is increasingly prevalent, yet it must remain a diagnosis of exclusion.
The combination of neurological features, haemolytic indices, and disproportionately low alkaline phosphatase values represented critical diagnostic signals. Importantly, the reduction in ceruloplasmin was modest, highlighting the limited value of isolated testing strategies.
Delayed recognition permitted disease progression to partially irreversible neurological injury and advanced hepatic dysfunction despite the availability of effective disease-modifying therapy.
Take-home message
- Think Wilson: In young adults, persistent aminotransferase elevation without a clear explanation should always prompt consideration of Wilson disease — even when abnormalities are mild and the clinical picture appears reassuring.
- Look at ALP: Routine liver tests may conceal critical diagnostic clues. A disproportionately low alkaline phosphatase level relative to bilirubin, particularly when associated with haemolytic features, represents an important warning signal for disorders of copper metabolism.
- Beyond Steatosis: Hepatic steatosis is common, but in young patients it must remain a diagnosis of exclusion. The presence of fatty liver should not lead to diagnostic closure when clinical or biochemical inconsistencies suggest alternative or rarer conditions.
- Avoid diagnostic closure: Common diagnoses explain common findings, but rare diseases often hide behind them. Persistent discordance between clinical features and routine investigations should always justify renewed diagnostic curiosity.
Wilson disease in clinical practice
Wilson disease is a rare autosomal recessive disorder of copper metabolism caused by pathogenic variants in the ATP7B gene, resulting in impaired biliary copper excretion and progressive copper accumulation. The estimated prevalence is approximately 1 in 30.000 individuals, with a carrier frequency of roughly 1 in 90, although regional variability exists. Because of underrecognition and phenotypic heterogeneity, true prevalence may be higher than traditionally reported.
Clinical presentation typically occurs between the first and fourth decades of life, most commonly during adolescence or young adulthood. Hepatic manifestations often predominate in children and younger patients, whereas neurological and neuropsychiatric features are more frequently observed in adolescents and adults. However, age at diagnosis is highly variable, and delayed recognition remains common, particularly in patients with mild or non-specific early findings.
Diagnosis requires a composite assessment integrating biochemical markers (including ceruloplasmin and 24-hour urinary copper excretion), ophthalmological evaluation, and genetic testing. No single parameter is sufficient to exclude the disease. Early detection is essential, as timely initiation of copper-lowering therapy can prevent irreversible hepatic and neurological injury, whereas delayed diagnosis may result in progressive organ dysfunction and permanent neurological sequelae.
- European Association for the Study of the Liver. EASL-ERN Clinical Practice Guidelines on Wilson's disease. J Hepatol. 2025 Feb 22:S0168-8278(24)02706-5. doi: 10.1016/j.jhep.2024.11.007. Epub ahead of print. PMID: 40089450.
- Członkowska A, Litwin T, Dusek P, Ferenci P, Lutsenko S, Medici V, Rybakowski JK, Weiss KH, Schilsky ML. Wilson disease. Nat Rev Dis Primers. 2018 Sep 6;4(1):21. doi: 10.1038/s41572-018-0018-3. PMID: 30190489; PMCID: PMC6416051.
- Shribman S, Poujois A, Bandmann O, Czlonkowska A, Warner TT. Wilson's disease: update on pathogenesis, biomarkers and treatments. J Neurol Neurosurg Psychiatry. 2021 Oct;92(10):1053-1061. doi: 10.1136/jnnp-2021-326123. Epub 2021 Aug 2. PMID: 34341141.
- Orphanet. Wilson disease. Orphanet Rare Disease resource.