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Wilson's Disease

VERSION: 1.1 CREATED: January 30, 2026 REVISED: January 30, 2026 STATUS: Approved


DIAGNOSIS: Wilson's Disease (Hepatolenticular Degeneration)

ICD-10: E83.01 (Wilson's disease), E83.09 (Other disorders of copper metabolism), G25.5 (Other chorea — for movement disorder presentation), G25.2 (Other specified forms of tremor — for tremor-predominant presentation), F06.8 (Other specified mental disorders due to known physiological condition — psychiatric presentation)

CPT CODES: 82390 (Serum ceruloplasmin), 82525 (Serum copper (total)), 85025 (CBC with differential), 80053 (CMP (BMP + LFTs)), 82248 (Direct and indirect bilirubin), 85610 (PT/INR), 85045 (Reticulocyte count), 86880 (Direct Coombs test (DAT)), 83010 (Haptoglobin), 83615 (LDH), 81003 (Urinalysis), 84100 (Phosphorus), 84550 (Uric acid), 82947 (Blood glucose), 81406 (ATP7B gene sequencing), 85060 (Peripheral blood smear), 82140 (Ammonia level), 83605 (Lactate), 82105 (Alpha-fetoprotein (AFP)), 80074 (Hepatitis panel (HAV, HBV, HCV)), 86235 (ANA, anti-smooth muscle antibody), 83540 (Iron studies), 84443 (TSH), 82607 (Vitamin B12), 86255 (Paraneoplastic antibody panel), 77080 (Bone densitometry (DEXA)), 70551 (MRI brain without contrast), 70553 (MRI brain with contrast), 76700 (Abdominal ultrasound with Doppler), 71046 (Chest X-ray), 74178 (CT abdomen with contrast), 74183 (MRI abdomen (liver)), 91200 (FibroScan (transient elastography)), 43239 (Upper endoscopy (EGD)), 93000 (ECG (12-lead)), 93306 (Echocardiogram), 95816 (EEG), 78608 (PET brain (FDG)), 76390 (MR spectroscopy (brain)), 78830 (DaTscan), 47000 (Liver biopsy)

SYNONYMS: Wilson disease, WD, hepatolenticular degeneration, progressive lenticular degeneration, Westphal-Strümpell pseudosclerosis, copper storage disease, ATP7B deficiency, hepatocerebral degeneration, Wilson's, copper overload disease, hepatolenticular syndrome, Kinnier Wilson disease

SCOPE: Diagnosis and management of Wilson's disease in adolescents and adults presenting with neurologic, hepatic, or psychiatric manifestations. Covers diagnostic workup (ceruloplasmin, 24-hour urine copper, slit-lamp exam, hepatic copper, genetic testing), chelation therapy (D-penicillamine, trientine), zinc maintenance therapy, liver transplant evaluation, symptomatic management of neurological symptoms (dystonia, tremor, parkinsonism, psychiatric), and family screening. Excludes other copper metabolism disorders (Menkes disease), isolated non-Wilson hepatic cirrhosis, and drug-induced movement disorders.


DEFINITIONS: - Wilson's Disease (WD): Autosomal recessive disorder caused by mutations in the ATP7B gene (chromosome 13q14.3), resulting in defective copper excretion into bile and incorporation into ceruloplasmin, leading to toxic copper accumulation in liver, brain, cornea, kidneys, and other organs - Kayser-Fleischer (KF) Rings: Golden-brown deposits of copper in the Descemet membrane of the cornea; detected by slit-lamp exam; present in >95% of neurologic WD and ~50% of hepatic-only WD - Wing-Beating Tremor: Coarse, irregular, proximal tremor elicited with arms outstretched and elbows flexed; characteristic (though not pathognomonic) of WD - Ceruloplasmin: Copper-carrying glycoprotein synthesized in the liver; typically low (<20 mg/dL) in WD but can be normal in 5-15% of neurologic WD - Free (Non-ceruloplasmin-bound) Copper: Calculated as total serum copper minus ceruloplasmin-bound copper; elevated in WD (>25 mcg/dL) - Leipzig Score: Diagnostic scoring system for WD incorporating KF rings, neurologic symptoms, ceruloplasmin, Coombs-negative hemolytic anemia, hepatic copper, urinary copper, and mutation analysis (score ≥4 = established diagnosis)


KEY CLINICAL FEATURES:

Neurologic Manifestations (40-50% of presentations): - Movement disorders: Dystonia (most common neurologic feature), tremor (resting, postural, wing-beating), parkinsonism, choreoathetosis, athetosis - Bulbar symptoms: Dysarthria (most common initial neurologic symptom), dysphagia, drooling, dysphonia - Gait disturbance: Ataxia, postural instability - Other: Seizures (rare, 6%), risus sardonicus (fixed grin from facial dystonia)

Hepatic Manifestations (40-50% of presentations): - Asymptomatic transaminase elevation - Chronic hepatitis - Cirrhosis (compensated or decompensated) - Fulminant hepatic failure (with Coombs-negative hemolytic anemia)

Psychiatric Manifestations (30-40%, often earliest): - Depression, anxiety - Personality change, irritability - Psychosis (rare) - Cognitive decline, declining school/work performance - Behavioral disinhibition

Other Manifestations: - Ophthalmologic: KF rings, sunflower cataracts - Hematologic: Coombs-negative hemolytic anemia - Renal: Fanconi syndrome, renal tubular acidosis, nephrolithiasis - Musculoskeletal: Osteoporosis, arthropathy - Cardiac: Cardiomyopathy (rare)


PRIORITY KEY: STAT = Immediate | URGENT = Within hours | ROUTINE = Standard | EXT = Extended/atypical cases | - = Not applicable to this setting

═══════════════════════════════════════════════════════════════ SECTION A: ACTION ITEMS ═══════════════════════════════════════════════════════════════

1. LABORATORY WORKUP

1A. Essential/Core Labs

Test ED HOSP OPD ICU Rationale Target Finding
Serum ceruloplasmin (CPT 82390) URGENT STAT ROUTINE STAT Low in 85-95% of WD; screening test; may be normal in acute liver failure or inflammation <20 mg/dL (often <10 mg/dL in WD); normal does NOT exclude WD
Serum copper (total) (CPT 82525) URGENT STAT ROUTINE STAT Low-normal total copper (paradoxically) but elevated free copper in WD Total copper low-normal (40-80 mcg/dL); calculate free copper
24-hour urine copper (CPT 82525) - STAT ROUTINE STAT Gold standard screening; elevated in WD; >100 mcg/24h suggestive; >40 mcg/24h in children >100 mcg/24h (often >200 in symptomatic WD); normal <40 mcg/24h
CBC with differential (CPT 85025) STAT STAT ROUTINE STAT Coombs-negative hemolytic anemia; baseline; thrombocytopenia (hypersplenism) Hemolytic anemia, thrombocytopenia suggest WD
CMP (BMP + LFTs) (CPT 80053) STAT STAT ROUTINE STAT AST/ALT elevation; synthetic function (albumin, bilirubin); renal function Elevated AST/ALT (often AST > ALT); low albumin; elevated bilirubin in advanced disease
Direct and indirect bilirubin (CPT 82248) STAT STAT ROUTINE STAT Elevated indirect bilirubin in hemolysis; elevated direct in hepatic disease Elevated (especially indirect if hemolysis)
PT/INR (CPT 85610) STAT STAT ROUTINE STAT Hepatic synthetic function; coagulopathy in liver failure Elevated INR in advanced disease; INR >1.5 suggests significant hepatic dysfunction
Reticulocyte count (CPT 85045) URGENT STAT ROUTINE STAT Elevated in hemolytic anemia Elevated (>2%) suggests active hemolysis
Direct Coombs test (DAT) (CPT 86880) URGENT STAT ROUTINE STAT NEGATIVE in WD hemolysis (Coombs-negative hemolytic anemia is classic) Negative (positive Coombs suggests other etiology)
Haptoglobin (CPT 83010) URGENT STAT ROUTINE STAT Low/undetectable in hemolysis Low or undetectable in active hemolysis
LDH (CPT 83615) URGENT STAT ROUTINE STAT Elevated in hemolysis Elevated in hemolysis
Urinalysis (CPT 81003) STAT STAT ROUTINE STAT Renal tubular dysfunction (glycosuria, proteinuria, aminoaciduria — Fanconi syndrome) Glycosuria with normal serum glucose; proteinuria
Phosphorus (CPT 84100) STAT STAT ROUTINE STAT Hypophosphatemia from renal tubular loss Low (Fanconi syndrome)
Uric acid (CPT 84550) - ROUTINE ROUTINE ROUTINE Low uric acid suggests renal tubular dysfunction Low (<2 mg/dL suggests renal copper toxicity)
Blood glucose (CPT 82947) STAT STAT ROUTINE STAT Baseline; glycosuria with normal glucose suggests Fanconi Normal (but glycosuria present = renal tubular)

1B. Extended Workup (Second-line)

Test ED HOSP OPD ICU Rationale Target Finding
Free (non-ceruloplasmin-bound) copper (calculated) - STAT ROUTINE STAT Calculated: total serum copper (mcg/dL) minus (3 x ceruloplasmin [mg/dL]); elevated in WD >25 mcg/dL (often >50 in symptomatic WD); most specific biochemical marker
Penicillamine challenge test (24-hour urine copper after 500 mg penicillamine x 2 doses) - ROUTINE ROUTINE - Used in equivocal cases, especially pediatric; less validated in adults >1600 mcg/24h highly suggestive of WD (>25 mcmol/24h)
ATP7B gene sequencing (CPT 81406) - ROUTINE ROUTINE - Definitive diagnosis; identifies pathogenic variants; essential for family screening Two pathogenic variants = confirmed WD; one variant + clinical findings = probable WD
Peripheral blood smear (CPT 85060) URGENT STAT ROUTINE STAT Schistocytes, spherocytes in hemolytic anemia Fragmented RBCs, polychromasia
Ammonia level (CPT 82140) STAT STAT - STAT Hepatic encephalopathy assessment in liver failure Normal <35 mcmol/L; elevated in hepatic failure
Lactate (CPT 83605) STAT STAT - STAT Tissue hypoperfusion in fulminant hepatic failure Elevated in liver failure
Alpha-fetoprotein (AFP) (CPT 82105) - ROUTINE ROUTINE - HCC screening in cirrhotic patients Normal; elevated suggests HCC
Hepatitis panel (HAV, HBV, HCV) (CPT 80074) - ROUTINE ROUTINE - Exclude concurrent viral hepatitis Negative
ANA, anti-smooth muscle antibody (CPT 86235) - ROUTINE ROUTINE - Exclude autoimmune hepatitis (may coexist or mimic) Negative or low titer
Iron studies (CPT 83540) - ROUTINE ROUTINE - Exclude hemochromatosis in hepatic presentation Normal ferritin and transferrin saturation
TSH (CPT 84443) - ROUTINE ROUTINE - Thyroid dysfunction can present with tremor/psychiatric symptoms Normal
Vitamin B12 (CPT 82607) - ROUTINE ROUTINE - B12 deficiency can cause neuropsychiatric symptoms Normal

1C. Rare/Specialized (Refractory or Atypical)

Test ED HOSP OPD ICU Rationale Target Finding
Hepatic copper concentration (liver biopsy) (CPT 82525) - ROUTINE ROUTINE - Gold standard for diagnosis; required when non-invasive testing equivocal; >250 mcg/g dry weight diagnostic >250 mcg/g dry weight (normal <50 mcg/g); values 50-250 can be seen in heterozygotes or cholestatic disease
Radioactive copper incorporation study (⁶⁴Cu or ⁶⁷Cu) - EXT EXT - Measures copper incorporation into ceruloplasmin over 48h; absent second peak in WD; rarely available Absent secondary rise at 48h (failure of copper incorporation into ceruloplasmin)
Relative exchangeable copper (REC) ratio - EXT EXT - Emerging biomarker; ratio of exchangeable copper to total copper; highly sensitive/specific >18.5% diagnostic of WD
Paraneoplastic antibody panel (CPT 86255) - EXT EXT - If atypical neurologic features not explained by WD alone Negative
Heavy metal screen (lead, mercury, arsenic) - EXT EXT - Toxic metal exposure can cause neuropsychiatric symptoms Normal
Urine amino acids - EXT EXT - Fanconi syndrome confirmation (generalized aminoaciduria) Elevated (generalized aminoaciduria in Fanconi)
Bone densitometry (DEXA) (CPT 77080) - - ROUTINE - Osteoporosis screening; copper toxicity causes bone loss T-score > -2.5

2. DIAGNOSTIC IMAGING & STUDIES

2A. Essential/First-line

Study ED HOSP OPD ICU Timing Target Finding Contraindications
Slit-lamp examination (ophthalmology) URGENT STAT ROUTINE STAT At initial evaluation; do NOT rely on naked-eye exam Kayser-Fleischer rings (present in >95% neurologic WD, ~50% hepatic-only); sunflower cataracts None
MRI brain without contrast (CPT 70551) URGENT URGENT ROUTINE URGENT Within 24-48h if neurologic symptoms T2/FLAIR hyperintensity in basal ganglia (putamen — "face of the giant panda" sign on midbrain), thalamus, caudate, brainstem, cerebellum, white matter; T1 hyperintensity (copper/manganese deposition); cerebral atrophy Pacemaker, metallic implants
MRI brain with contrast (CPT 70553) - ROUTINE ROUTINE - If additional characterization needed Rarely enhances; helps exclude other pathology Contrast allergy, GFR <30
Abdominal ultrasound with Doppler (CPT 76700) URGENT STAT ROUTINE STAT At diagnosis Hepatomegaly, splenomegaly, cirrhotic changes, portal hypertension, ascites None significant
Chest X-ray (CPT 71046) URGENT ROUTINE - STAT On admission for fulminant hepatic failure; baseline for ICU Aspiration, atelectasis, pleural effusion, pulmonary edema None significant

2B. Extended

Study ED HOSP OPD ICU Timing Target Finding Contraindications
CT abdomen with contrast (CPT 74178) - ROUTINE ROUTINE - If ultrasound equivocal; HCC screening Cirrhosis, portal hypertension, hepatic lesions Contrast allergy, renal impairment
MRI abdomen (liver) (CPT 74183) - ROUTINE ROUTINE - Liver characterization; iron vs copper deposition Hepatic signal abnormalities; excludes HCC Per MRI
FibroScan (transient elastography) (CPT 91200) - - ROUTINE - Non-invasive fibrosis assessment Liver stiffness <7.1 kPa = minimal fibrosis; >12.5 kPa = cirrhosis Ascites, obesity (limited), pacemaker
Upper endoscopy (EGD) (CPT 43239) - ROUTINE ROUTINE - Variceal screening in cirrhotic patients No varices; if present, grade and treat Active GI perforation
ECG (12-lead) (CPT 93000) URGENT ROUTINE ROUTINE URGENT Baseline; cardiac involvement Arrhythmias, conduction abnormalities None
Echocardiogram (CPT 93306) - ROUTINE ROUTINE - If cardiac symptoms or cardiomyopathy suspected Normal function; WD cardiomyopathy rare None significant
NCS/EMG (CPT 95907-95913 / 95886) - - ROUTINE - If peripheral neuropathy suspected Rule out concurrent neuropathy None significant
EEG (CPT 95816) URGENT ROUTINE - URGENT If seizures suspected or encephalopathy Generalized slowing in encephalopathy; epileptiform discharges None

2C. Rare/Specialized

Study ED HOSP OPD ICU Timing Target Finding Contraindications
PET brain (FDG) (CPT 78608) - - EXT - If atypical features or diagnostic uncertainty Hypometabolism in basal ganglia, cerebellum Per PET
MR spectroscopy (brain) (CPT 76390) - EXT EXT - Research; metabolic characterization Reduced NAA (neuronal loss) in basal ganglia Per MRI
DaTscan (CPT 78830) - - EXT - Differentiate WD parkinsonism from idiopathic PD if uncertain May show reduced uptake (presynaptic dopaminergic loss); less specific in WD Pregnancy, iodine allergy
Liver biopsy (CPT 47000) - ROUTINE ROUTINE - When non-invasive testing equivocal; hepatic copper quantification Copper >250 mcg/g dry weight = diagnostic; histology shows steatosis, hepatitis, cirrhosis Coagulopathy (INR >1.5), ascites (use transjugular), thrombocytopenia

3. TREATMENT

3A. Acute/Emergent

Treatment Route Indication Dosing Contraindications Monitoring ED HOSP OPD ICU
N-acetylcysteine (NAC) IV Fulminant hepatic failure; hepatoprotection 150 mg/kg :: IV :: loading :: 150 mg/kg IV over 1h, then 50 mg/kg over 4h, then 100 mg/kg over 16h (standard NAC protocol for liver failure) None absolute Anaphylactoid reaction, bronchospasm; monitor LFTs, INR STAT STAT - STAT
Fresh frozen plasma (FFP) IV Coagulopathy in fulminant hepatic failure 10-15 mL/kg :: IV :: PRN :: 10-15 mL/kg IV; target INR <1.5 for procedures; use as bridge to transplant Volume overload; IgA deficiency INR, fibrinogen, volume status STAT STAT - STAT
Vitamin K (phytonadione) IV Coagulopathy; hepatic synthetic failure 10 mg :: IV :: daily :: 10 mg IV daily x 3 days; limited efficacy in WD fulminant failure (hepatocyte loss) Allergy INR response STAT STAT - STAT
Lactulose PO Hepatic encephalopathy 30 mL :: PO :: TID :: 30 mL PO TID; titrate to 3-4 bowel movements/day Bowel obstruction Mental status, ammonia, stool output STAT STAT - STAT
Rifaximin PO Hepatic encephalopathy (adjunct to lactulose) 550 mg :: PO :: BID :: 550 mg PO BID; add if lactulose alone insufficient C. difficile (relative) Mental status, hepatic function - ROUTINE - ROUTINE
Albumin (25%) IV Ascites management; hepatorenal syndrome 1 g/kg :: IV :: daily :: 1 g/kg/day (max 100 g); for large-volume paracentesis or hepatorenal syndrome Heart failure Volume status, serum albumin - STAT - STAT
Platelet transfusion IV Thrombocytopenia with active bleeding or pre-procedure Dose per protocol :: IV :: PRN :: 1 unit apheresis platelets; target >50K for procedures, >10K if no bleeding ITP, TTP (relative) Platelet count post-transfusion STAT STAT - STAT
Plasmapheresis/Plasma exchange Extracorporeal Fulminant WD with severe hemolysis; removes free copper 1-1.5 plasma volumes :: Extracorporeal :: daily :: Daily exchange x 3-5 days; rapidly reduces free copper Hemodynamic instability, severe sepsis Copper levels, hemolysis markers, coagulation - STAT - STAT
DVT prophylaxis: Enoxaparin SC Immobilized hospitalized patients 40 mg :: SC :: daily :: 40 mg SC daily; hold if INR >1.5 or platelets <50K or active bleeding Active bleeding, severe coagulopathy, CrCl <30 (use UFH) Platelets, signs of bleeding - ROUTINE - ROUTINE

3B. Symptomatic Treatments

Treatment Route Indication Dosing Contraindications Monitoring ED HOSP OPD ICU
Baclofen PO Dystonia, spasticity 5 mg :: PO :: TID :: Start 5 mg TID; titrate by 5 mg/dose every 3 days; target 30-80 mg/day divided TID; max 80 mg/day Severe renal impairment; abrupt withdrawal risk Sedation, weakness; do NOT stop abruptly (withdrawal seizures) - ROUTINE ROUTINE ROUTINE
Trihexyphenidyl PO Dystonia (focal or generalized) 1 mg :: PO :: daily :: Start 1 mg daily; increase by 1 mg every 3-5 days; target 6-15 mg/day divided TID; max 15 mg/day Glaucoma, urinary retention, dementia, age >65 Confusion, dry mouth, urinary retention, cognitive impairment - ROUTINE ROUTINE -
Levodopa/Carbidopa PO Parkinsonism (rigidity, bradykinesia) 100 mg :: PO :: TID :: Start 25/100 mg TID; titrate by 25/100 mg q1-2 weeks; limited efficacy in WD parkinsonism (response variable) Narrow-angle glaucoma Dyskinesia, nausea, orthostatic hypotension; often poor response in WD - ROUTINE ROUTINE -
Clonazepam PO Tremor, dystonia, chorea, sleep disturbance 0.5 mg :: PO :: BID :: Start 0.5 mg BID; titrate by 0.5 mg q3-5 days; max 4 mg/day Respiratory depression, severe hepatic disease, addiction risk Sedation, respiratory depression, dependence; adjust dose in hepatic impairment - ROUTINE ROUTINE ROUTINE
Botulinum toxin (OnabotulinumtoxinA) IM Focal dystonia (cervical, oromandibular, limb) Dose per target :: IM :: q3 months :: Site-specific dosing: cervical dystonia 100-300 units; limb dystonia 50-200 units per muscle; q12 weeks Infection at injection site, myasthenia gravis Weakness at injection site, dysphagia (cervical injections) - - ROUTINE -
Propranolol PO Tremor (postural/action component) 20 mg :: PO :: BID :: Start 20 mg BID; titrate to 120-240 mg/day; limited efficacy for wing-beating tremor Asthma, COPD, bradycardia, decompensated heart failure HR, BP - ROUTINE ROUTINE -
Sertraline PO Depression, anxiety (common in WD) 25 mg :: PO :: daily :: Start 25 mg daily; titrate to 100-200 mg/day MAOI use; caution with hepatic impairment (reduce dose) Hepatic function (use lower doses in liver disease); suicidality in young adults - ROUTINE ROUTINE -
Quetiapine PO Psychosis, agitation, behavioral disturbance 12.5 mg :: PO :: QHS :: Start 12.5 mg QHS; titrate cautiously to 50-200 mg/day; avoid typical antipsychotics (worsen movement disorder) QTc prolongation; avoid typical antipsychotics (D2 blockade worsens dystonia/parkinsonism) QTc, metabolic panel, sedation; use ONLY atypical antipsychotics - ROUTINE ROUTINE ROUTINE
Gabapentin PO Neuropathic pain, tremor (adjunctive) 300 mg :: PO :: TID :: Start 300 mg QHS; increase by 300 mg/day every 3 days; target 900-1800 mg/day TID Severe renal impairment (dose adjust) Sedation, dizziness - ROUTINE ROUTINE -
Omeprazole PO GI protection during chelation therapy 20 mg :: PO :: daily :: 20 mg PO daily; take 30 min before chelator None absolute Long-term: B12, Mg levels - ROUTINE ROUTINE -

3C. Second-line/Refractory

Treatment Route Indication Dosing Contraindications Monitoring ED HOSP OPD ICU
Liver transplantation evaluation Referral Fulminant hepatic failure; decompensated cirrhosis unresponsive to chelation; severe neurologic WD refractory to 6+ months chelation (controversial) Refer to transplant center :: - :: - :: Urgent evaluation in fulminant WD (New Wilson Index ≥11 = transplant); King's Wilson criteria; MELD score >15 or acute liver failure Absolute contraindications per transplant center protocol Pre-transplant workup; transplant corrects metabolic defect (no lifelong chelation needed post-transplant) - URGENT ROUTINE STAT
Continuous venovenous hemofiltration (CVVH) Extracorporeal Fulminant WD with renal failure or massive hemolysis; bridge to transplant Per ICU protocol :: Extracorporeal :: continuous :: Continuous copper removal; bridge therapy to transplant N/A (life-saving in ICU) Copper levels, hemodynamics, electrolytes - - - STAT
Molecular adsorbent recirculating system (MARS) Extracorporeal Fulminant WD; albumin dialysis removes copper Per protocol :: Extracorporeal :: per session :: Albumin dialysis; available at specialized centers; bridge to transplant Hemodynamic instability Copper levels, bilirubin, coagulation, hemodynamics - EXT - EXT
Tetrathiomolybdate (ammonium or bis-choline) PO Investigational chelator; neurologic WD (may have less neurologic worsening than penicillamine) 20 mg :: PO :: TID :: 20 mg TID with meals + 20 mg TID between meals (total 120 mg/day); investigational — available through clinical trials or compassionate use Severe hepatic failure; not yet widely approved CBC (bone marrow suppression), LFTs, copper levels, iron studies (iron deficiency from over-decoppering) - EXT EXT -

3D. Disease-Modifying Therapies (Chelation & Zinc)

Treatment Route Indication Dosing Pre-Treatment Requirements Contraindications Monitoring ED HOSP OPD ICU
D-Penicillamine (Cuprimine) PO First-line chelation (symptomatic WD — hepatic or neurologic); removes copper via renal excretion 250 mg :: PO :: daily :: Start 250-500 mg/day; increase by 250 mg every 4-7 days; target 1000-1500 mg/day divided BID-QID; take on empty stomach (1h before or 2h after meals); SLOW titration reduces neurologic worsening risk Baseline: CBC, UA, CMP, 24h urine copper; pyridoxine 25 mg/day (penicillamine depletes B6); pregnancy test in women of childbearing age Penicillin allergy (may cross-react); pregnancy (teratogenic — switch to zinc); lupus-like syndrome; prior severe adverse reaction CBC weekly x1 month then monthly x6 months then q3 months; UA for proteinuria every 2 weeks x3 months then monthly; LFTs monthly; 24h urine copper q3-6 months (target 200-500 mcg/24h on treatment); watch for neurologic WORSENING in first 6-8 weeks (occurs in 10-50%) - STAT ROUTINE STAT
Trientine (Syprine, TETA) PO Alternative first-line chelator; preferred if penicillamine intolerant or neurologic presentation (lower risk of neurologic worsening); removes copper via renal excretion 250 mg :: PO :: BID :: Start 500-750 mg/day divided BID-TID; target 750-1500 mg/day; take on empty stomach (1h before or 2h after meals); available as trientine dihydrochloride or trientine tetrahydrochloride (Cuvrior — different dosing) Baseline: CBC, CMP, 24h urine copper; iron studies (trientine chelates iron); pregnancy test Pregnancy (teratogenic — switch to zinc); iron deficiency anemia (monitor closely) CBC monthly x6 months then q3 months; LFTs monthly x6 months then q3 months; 24h urine copper q3-6 months (target 200-500 mcg/24h); iron studies q6 months (sideropenia risk); watch for neurologic worsening (less common than penicillamine, ~10-15%) - STAT ROUTINE STAT
Zinc acetate (Galzin) PO Maintenance therapy (after initial decoppering with chelator); mild/presymptomatic WD; pregnancy (safest option); family members with pre-clinical WD 50 mg :: PO :: TID :: 50 mg elemental zinc TID (taken as zinc acetate 150 mg TID); take on empty stomach separated from meals by ≥1 hour; separate from chelator by ≥2 hours if used concurrently Baseline: CBC, CMP, 24h urine copper, serum zinc level None absolute; GI intolerance common (nausea); do NOT use as sole therapy in symptomatic/severe WD (too slow onset) 24h urine copper q3-6 months (target <75 mcg/24h on zinc monotherapy); serum zinc level q3-6 months (target 125-150 mcg/dL); 24h urine zinc (confirms compliance: >2 mg/24h); LFTs q3-6 months; CBC q6 months (zinc-induced copper deficiency can cause neutropenia) - ROUTINE ROUTINE -
Zinc sulfate (alternative zinc formulation) PO Alternative to zinc acetate for maintenance therapy; more GI side effects but less expensive 220 mg :: PO :: TID :: 220 mg zinc sulfate TID (contains ~50 mg elemental zinc per 220 mg); take on empty stomach separated from meals by ≥1 hour; separate from chelator by ≥2 hours if used concurrently Baseline: CBC, CMP, 24h urine copper, serum zinc level GI intolerance common (nausea, gastric irritation); do NOT use as sole therapy in symptomatic/severe WD (too slow onset) 24h urine copper q3-6 months (target <75 mcg/24h on zinc monotherapy); serum zinc level q3-6 months (target 125-150 mcg/dL); 24h urine zinc (confirms compliance: >2 mg/24h); LFTs q3-6 months; CBC q6 months (zinc-induced copper deficiency can cause neutropenia) - ROUTINE ROUTINE -
Pyridoxine (Vitamin B6) PO Required supplement with D-penicillamine therapy (penicillamine depletes B6) 25 mg :: PO :: daily :: 25 mg PO daily; co-administer with penicillamine None None Clinical assessment; peripheral neuropathy symptoms - ROUTINE ROUTINE -

4. OTHER RECOMMENDATIONS

4A. Referrals & Consults

Recommendation ED HOSP OPD ICU Indication
Hepatology consultation STAT STAT ROUTINE STAT All WD patients; liver disease staging, transplant evaluation, chelation management
Neurology consultation URGENT STAT ROUTINE STAT All neurologic WD; movement disorder management, monitoring for chelation-related worsening
Movement disorder specialist - ROUTINE ROUTINE - Dystonia, tremor, parkinsonism management; botulinum toxin injections
Ophthalmology (slit-lamp exam) URGENT STAT ROUTINE - ALL suspected WD — Kayser-Fleischer ring assessment; sunflower cataract screening
Genetics / Genetic counselor - ROUTINE ROUTINE - ATP7B testing confirmation, family screening, reproductive counseling
Transplant hepatology/surgery - STAT ROUTINE STAT Fulminant hepatic failure, decompensated cirrhosis, New Wilson Index ≥11
Psychiatry - ROUTINE ROUTINE - Depression, psychosis, personality change, behavioral disturbance (common in WD)
Neuropsychology - - ROUTINE - Baseline cognitive assessment; monitor for cognitive decline
Speech-language pathology (SLP) - ROUTINE ROUTINE - Dysarthria, dysphagia evaluation and management
Physical therapy (PT) - ROUTINE ROUTINE - Dystonia management, gait training, falls prevention
Occupational therapy (OT) - ROUTINE ROUTINE - ADL assistance, handwriting aids, adaptive equipment
Nutrition / Dietitian - ROUTINE ROUTINE - Low-copper diet counseling; hepatic diet if cirrhotic
Social work - ROUTINE ROUTINE - Chronic disease support, medication access (chelators are expensive), disability resources

4B. Patient/Family Instructions

Recommendation ED HOSP OPD ICU
Wilson's disease is a lifelong condition requiring CONTINUOUS treatment — never stop chelation or zinc without physician guidance - STAT ROUTINE -
Take chelation medication (penicillamine or trientine) on an EMPTY STOMACH — 1 hour before or 2 hours after meals - ROUTINE ROUTINE -
If taking zinc, separate from chelator by at least 2 hours; take zinc between meals - ROUTINE ROUTINE -
Return to ED if: worsening jaundice, confusion, vomiting blood, black stools, new neurologic symptoms, worsening speech or swallowing, severe abdominal pain STAT ROUTINE ROUTINE -
Report ANY new neurologic symptoms during the first 2-3 months of chelation (worsening is possible and requires dose adjustment) - STAT ROUTINE -
Avoid HIGH-COPPER FOODS during initial treatment: liver/organ meats, shellfish (especially lobster, oysters), chocolate, mushrooms, nuts, dried fruits, soy products - ROUTINE ROUTINE -
Avoid drinking water through copper pipes (consider water testing; use filter or bottled water if copper pipes present) - ROUTINE ROUTINE -
Alcohol is STRICTLY prohibited — causes additional liver damage - ROUTINE ROUTINE -
Women of childbearing age: pregnancy requires switching from chelator to zinc (chelators are teratogenic); discuss family planning with physician BEFORE conception - ROUTINE ROUTINE -
ALL first-degree relatives (siblings, children, parents) MUST be screened for Wilson's disease — siblings have 25% risk - ROUTINE ROUTINE -
Carry a medical alert card/bracelet indicating Wilson's disease and current medications - ROUTINE ROUTINE -
Regular follow-up is ESSENTIAL — blood and urine tests needed every 3-6 months lifelong - ROUTINE ROUTINE -

4C. Lifestyle & Prevention

Recommendation ED HOSP OPD ICU
Low-copper diet during initial decoppering phase (avoid >1 mg copper/day); can liberalize slightly once stable on treatment - ROUTINE ROUTINE -
Regular exercise as tolerated; may improve dystonia and mood - ROUTINE ROUTINE -
Strict alcohol avoidance (lifelong — hepatotoxic) - ROUTINE ROUTINE -
Smoking cessation (impairs liver function and overall health) - ROUTINE ROUTINE -
Hepatitis A and B vaccination if not immune (protect remaining liver function) - ROUTINE ROUTINE -
Avoid hepatotoxic medications (acetaminophen limit <2 g/day; avoid NSAIDs if cirrhotic; avoid statins if decompensated) - ROUTINE ROUTINE -
Genetic counseling for reproductive planning; autosomal recessive — both parents are carriers; 25% risk for each sibling - - ROUTINE -
Mental health support; depression and anxiety are common and treatable components of WD - ROUTINE ROUTINE -
Screen all first-degree relatives: ceruloplasmin, 24h urine copper, LFTs, slit-lamp exam, ATP7B genetic testing - - ROUTINE -

═══════════════════════════════════════════════════════════════ SECTION B: REFERENCE (Expand as Needed) ═══════════════════════════════════════════════════════════════

5. DIFFERENTIAL DIAGNOSIS

Alternative Diagnosis Key Distinguishing Features Tests to Differentiate
Autoimmune hepatitis Young women, positive ANA/SMA/anti-LKM, elevated IgG, no KF rings, no neurologic features; can coexist with WD ANA, SMA, IgG level, liver biopsy; ceruloplasmin, copper studies
Drug-induced parkinsonism Temporal relationship to dopamine-blocking medication, symmetric, resolves with drug withdrawal Medication review; ceruloplasmin normal; no KF rings
Idiopathic Parkinson's disease Asymmetric onset, rest tremor (pill-rolling), levodopa-responsive, age >50 typically, no hepatic involvement DaTscan; ceruloplasmin, 24h urine copper; slit-lamp (no KF rings)
Juvenile Huntington's disease Family history (AD), progressive chorea, behavioral changes, caudate atrophy on MRI Genetic testing (HTT CAG repeat); ceruloplasmin normal
Dystonia (primary/DYT) Isolated dystonia without hepatic features, family history, onset typically in limb, no KF rings Genetic testing (DYT1, etc.); ceruloplasmin normal; no hepatic involvement
Hemochromatosis Iron overload, skin bronzing, diabetes, arthropathy, hepatic disease, no neurologic copper features Iron studies (elevated ferritin, transferrin saturation); HFE gene; ceruloplasmin normal
Non-alcoholic fatty liver disease (NAFLD) Metabolic syndrome, obesity, insulin resistance, no neurologic features, no KF rings Metabolic workup; ceruloplasmin normal; liver biopsy (steatosis without copper)
Viral hepatitis (B, C) Exposure risk factors, positive viral serologies, no KF rings or neurologic features Hepatitis panel; ceruloplasmin normal
Alcoholic liver disease Alcohol use history, AST:ALT ratio >2, no KF rings, no neurologic copper features History; ceruloplasmin normal; liver biopsy
Neuroacanthocytosis Orofacial dyskinesia, chorea, self-mutilation, acanthocytes on blood smear Peripheral smear (acanthocytes); CK (elevated); ceruloplasmin normal
Menkes disease X-linked; presents in infancy (not adolescent/adult); kinky hair, seizures, failure to thrive Age of onset; genetic testing (ATP7A); ceruloplasmin very low but different gene
Pantothenate kinase-associated neurodegeneration (PKAN) "Eye of the tiger" sign on MRI (globus pallidus), dystonia, retinopathy MRI (iron deposition pattern differs from WD); PANK2 gene
Acquired hepatocerebral degeneration Chronic liver disease from ANY cause; parkinsonism, ataxia; MRI shows T1 basal ganglia signal (manganese) Underlying liver disease diagnosis; ceruloplasmin may be low from liver failure but 24h urine copper not markedly elevated; no ATP7B mutations
Multiple sclerosis Relapsing white matter disease, optic neuritis, spinal cord lesions; no hepatic involvement MRI brain/spine (demyelination); LP (oligoclonal bands); ceruloplasmin normal

6. MONITORING PARAMETERS

Parameter ED HOSP OPD ICU Frequency Target/Threshold Action if Abnormal
24-hour urine copper - STAT ROUTINE STAT q3-6 months on chelation; q6-12 months on zinc maintenance Chelation target: 200-500 mcg/24h; Zinc target: <75 mcg/24h Adjust chelator dose if outside range; <200 suggests over-chelation or non-compliance
Serum free (non-ceruloplasmin-bound) copper - STAT ROUTINE STAT q3-6 months <15 mcg/dL on treatment; negative values suggest over-treatment Reduce chelator if over-treated; increase if still elevated
LFTs (AST, ALT, bilirubin, albumin) STAT STAT ROUTINE STAT Monthly x6 months, then q3-6 months Normalizing or stable If worsening on treatment: assess compliance, consider dose adjustment, transplant evaluation
CBC with differential STAT STAT ROUTINE STAT Weekly x1 month (penicillamine), then monthly x6 months, then q3 months WBC >3000, platelets >100K, Hgb stable Penicillamine: hold if WBC <3000 or platelets <100K (bone marrow suppression); neutropenia on zinc (copper deficiency)
INR/PT STAT STAT ROUTINE STAT Monthly x6 months, then q3-6 months Normalizing (<1.3) Worsening INR suggests progressive liver disease; transplant evaluation if persistent
Urinalysis (for proteinuria) - ROUTINE ROUTINE - Every 2 weeks x3 months on penicillamine, then monthly No proteinuria (>1+ protein warrants investigation) Penicillamine nephrotoxicity: hold if >1 g/24h proteinuria; may require switch to trientine
Serum zinc level - ROUTINE ROUTINE - q3-6 months on zinc therapy 125-150 mcg/dL (therapeutic range) Low: assess compliance; High: reduce dose
24-hour urine zinc - - ROUTINE - q6-12 months >2 mg/24h confirms compliance Low suggests non-compliance
Neurologic exam (dystonia, tremor, speech, gait) URGENT STAT ROUTINE STAT Each visit; monthly during initial chelation Stable or improving; no new deficits If WORSENING in first 2-3 months: reduce chelator dose, consider switch to trientine or add zinc; persistent worsening → transplant evaluation
Slit-lamp exam (KF rings) - STAT ROUTINE - Annually on treatment Gradual fading of KF rings (may take 3-5 years) Persistence or reappearance suggests inadequate treatment or non-compliance
MRI brain - ROUTINE ROUTINE - Baseline; repeat at 6-12 months; then annually x3 years if neurologic Improving or stable signal abnormalities; resolving edema Worsening lesions suggest inadequate treatment or chelation-induced worsening
Hepatic fibrosis assessment (FibroScan or biomarkers) - - ROUTINE - Annually if cirrhotic Stable or improving stiffness Worsening fibrosis despite treatment: re-evaluate; transplant referral
Iron studies - ROUTINE ROUTINE - q6 months on trientine Normal ferritin and iron Trientine chelates iron → sideropenia; supplement iron if deficient (separate from trientine by 2h)
Bone density (DEXA) - - ROUTINE - Baseline; repeat q2 years T-score > -2.5 Treat osteoporosis; calcium/vitamin D supplementation
Depression/psychiatric screening - ROUTINE ROUTINE - Each visit Stable mood, no psychosis Psychiatric referral; adjust medications

7. DISPOSITION CRITERIA

Disposition Criteria
Discharge home Diagnosis established; chelation/zinc initiated and tolerated; stable neurologic exam; no hepatic decompensation; able to take oral medications; understands dietary restrictions and medication timing; follow-up arranged within 1-2 weeks
Admit to floor New diagnosis requiring inpatient workup; initiation of chelation therapy with monitoring; hepatic decompensation (ascites, jaundice, coagulopathy); moderate neurologic symptoms requiring inpatient management; inability to take oral medications
Admit to ICU Fulminant hepatic failure (encephalopathy, coagulopathy, renal failure); severe hemolytic crisis; status epilepticus; severe dystonic crisis with airway compromise; pending liver transplant evaluation
Transfer to liver transplant center Fulminant Wilson's disease (New Wilson Index ≥11); decompensated cirrhosis unresponsive to chelation; King's criteria met; MELD score >15 with progressive decline
Outpatient management Stable on chelation or zinc maintenance; routine monitoring labs; presymptomatic WD diagnosed through family screening; mild hepatic or neurologic disease responding to treatment
Inpatient rehabilitation Severe dystonia or parkinsonism affecting function; dysphagia requiring supervised feeding; gait instability requiring intensive PT

8. EVIDENCE & REFERENCES

Recommendation Evidence Level Source
Ceruloplasmin <20 mg/dL as screening test for WD Class II, Level B Ferenci et al., Liver Int 2003 PubMed: 14506258
24-hour urine copper >100 mcg as diagnostic criterion Class II, Level B Roberts & Schilsky, Hepatology 2008 (AASLD Practice Guidelines) PubMed: 18506894
D-Penicillamine as first-line chelation therapy Class II, Level B Walshe, Lancet 1956; Roberts & Schilsky, Hepatology 2008 PubMed: 18506894
Trientine as alternative first-line chelator with lower neurologic worsening risk Class II, Level B Walshe, Lancet 1982; Weiss et al., J Hepatol 2013 PubMed: 23485523
Zinc acetate for maintenance therapy Class II, Level B Brewer et al., Arch Neurol 1987; Czlonkowska et al., Ann Neurol 1996 PubMed: 8967753
Neurologic worsening occurs in 10-50% of patients starting D-penicillamine Class II, Level B Brewer et al., Arch Neurol 1994 PubMed: 7944997; Walshe & Yealland, J Neurol Neurosurg Psychiatry 1993 PubMed: 8505642
Liver transplantation for fulminant WD and decompensated cirrhosis Class I, Level B Arnon et al., Hepatology 2011 PubMed: 21254169; Dhawan et al., Gut 2005 PubMed: 15753547
New Wilson Index ≥11 predicts need for transplantation in pediatric fulminant WD Class II, Level B Dhawan et al., Gut 2005 PubMed: 15753547
Kayser-Fleischer rings present in >95% of neurologic WD Class II, Level B Walshe, Brain 1976; Ala et al., Lancet 2007 PubMed: 17276780
Leipzig Scoring System for WD diagnosis (score ≥4 = established) Class IIa, Level C Ferenci et al., Liver Int 2003 (8th International Meeting on Wilson Disease) PubMed: 14506258
ATP7B gene testing for definitive diagnosis and family screening Class I, Level B Loudianos & Gitlin, Semin Liver Dis 2000 PubMed: 11200483; EASL Clinical Practice Guidelines 2012 PubMed: 22276820
MRI brain findings: T2 hyperintensity in putamen ("face of giant panda" sign) Class II, Level C King et al., Mov Disord 1996 PubMed: 8866494; Sinha et al., AJNR 2006 PubMed: 16551993
Low-copper diet during initial decoppering phase Class III, Level C Expert consensus; EASL Clinical Practice Guidelines 2012 PubMed: 22276820
Plasmapheresis for acute hemolytic crisis in fulminant WD Class IIb, Level C Jhang et al., J Clin Apher 2007 PubMed: 17722038; Akyildiz et al., Ther Apher Dial 2004 PubMed: 15255163
Free (non-ceruloplasmin-bound) copper as monitoring parameter Class II, Level B Walshe, Ann Clin Biochem 2003 PubMed: 12880538; EASL Clinical Practice Guidelines 2012 PubMed: 22276820
Pregnancy management: switch to zinc; chelators are teratogenic Class II, Level C Brewer et al., J Lab Clin Med 2000 PubMed: 10638697; Tarnacka et al., Fertil Steril 2000 PubMed: 10685533
Family screening with ceruloplasmin, 24h urine copper, and ATP7B genotyping Class I, Level B EASL Clinical Practice Guidelines 2012 PubMed: 22276820; Roberts & Schilsky, Hepatology 2008 PubMed: 18506894
AASLD Practice Guidelines for Wilson Disease Class I (Guidelines) Roberts & Schilsky, Hepatology 2008 PubMed: 18506894; Schilsky, Clin Liver Dis 2017 PubMed: 28364808
EASL Clinical Practice Guidelines on Wilson's Disease Class I (Guidelines) European Association for the Study of the Liver, J Hepatol 2012 PubMed: 22276820
Trientine tetrahydrochloride (Cuvrior) as improved formulation Class II, Level B Weiss et al., Lancet Gastroenterol Hepatol 2022 PubMed: 35276119
Bis-choline tetrathiomolybdate for neurologic WD (phase 3 data) Class II, Level B Weiss et al., Lancet Gastroenterol Hepatol 2022 (WTX101-301 trial) PubMed: 35276119

NOTES

  • Wilson's disease is a TREATABLE cause of liver failure and neurologic decline — early diagnosis is critical
  • Consider WD in ANY patient <50 years with unexplained liver disease, movement disorder, or psychiatric illness
  • Ceruloplasmin alone misses 5-15% of cases; always obtain 24-hour urine copper and slit-lamp exam
  • The Leipzig Score integrates multiple tests for diagnostic certainty (score ≥4 = WD established)
  • D-penicillamine is effective but causes neurologic WORSENING in 10-50% of patients during initial weeks; many experts now prefer trientine as first-line for neurologic WD
  • Zinc monotherapy is too slow for symptomatic disease but is ideal for maintenance and presymptomatic patients
  • NEVER stop chelation/zinc abruptly — can precipitate fulminant hepatic failure or neurologic crisis
  • Avoid typical antipsychotics (haloperidol, etc.) — D2 blockade worsens dystonia and parkinsonism; use quetiapine or clozapine if antipsychotic needed
  • Fulminant Wilson's disease: Coombs-negative hemolytic anemia + acute liver failure + low ALP + low ALP-to-bilirubin ratio is classic triad; requires urgent transplant evaluation
  • All first-degree relatives MUST be screened; siblings have 25% risk of being affected
  • Liver transplantation CURES the metabolic defect — post-transplant patients do not need chelation
  • MRI brain may lag behind clinical improvement by months; initial worsening of MRI does not necessarily indicate treatment failure
  • When using combined chelation + zinc therapy, separate zinc from chelator by ≥2 hours (zinc blocks chelator absorption if given together); some experts use chelator + zinc simultaneously for initial treatment of symptomatic WD

CHANGE LOG

v1.1 (January 30, 2026) - Fixed ICD-10 codes: removed K74.3 (primary biliary cirrhosis — not a WD code) and G25.0 (essential tremor — not appropriate for WD tremor); added G25.2 (other specified forms of tremor) per C3, C4 - Removed "Combined chelation + zinc" row from Section 3D (used cross-references "Per individual agents"); moved combination note to NOTES section per C1, R1 - Made Zinc sulfate row fully self-contained: expanded Pre-Treatment, Contraindications, and Monitoring fields (previously "Same as zinc acetate") per C2, R2 - Updated Pyridoxine Monitoring from "None specific" to "Clinical assessment; peripheral neuropathy symptoms" and Contraindications from "None specific" to "None" per M5 - Standardized starting doses: Propranolol (20 mg), Sertraline (25 mg), Quetiapine (12.5 mg) — changed from ranges per R5, M2-M4 - Updated Plasmapheresis Route to "Extracorporeal" (was "-") per S1, R9 - Updated CVVH Route to "Extracorporeal" (was "IV") and MARS Route to "Extracorporeal" (was "-") per S2 - Updated Liver transplantation evaluation Route to "Referral" (was "-") per S2 - Added ICU column to Section 4B (Patient/Family Instructions) and Section 4C (Lifestyle & Prevention) tables per S3, S4 - Added Chest X-ray to Section 2A (Essential imaging) for ICU/fulminant hepatic failure patients per R6 - Added PubMed citation links to all 22 references in Section 8 per R7 - Added REVISED date to header metadata - Version bump from 1.0 to 1.1

v1.0 (January 30, 2026) - Initial template creation - Comprehensive diagnostic workup including Leipzig Score components - Chelation therapy with structured dosing (D-penicillamine, trientine, zinc) - Symptomatic management for dystonia, tremor, parkinsonism, psychiatric symptoms - Fulminant hepatic failure acute management - Liver transplant evaluation criteria - Family screening recommendations - 22 evidence-based references with author, journal, and year