Table 4.

Drugs used in treatment of Wilson disease.^3,7,17

Drug	Mode of action	Adverse effects	Dose	Monitoring adequacy
D-Penicillamine	Chelates copper and induces copper excretion	Early: Fever, rash, lymphadenopathy, cytopenias, proteinuria Late: Nephrotoxicity, lupus like syndrome, good Pasteur syndrome, bone marrow toxicity, dermatological toxicity (elastosis perforans serpiginosa) Very late: Nephrotoxicity, myasthenia gravis, polymyositis	Initial: Adult 750 mg–1500 mg BID—QID Children—20 mg/kg/d BID—QID Maintenance 15 mg/kg/day	24 h urinary copper Nonceruloplasmin bound copper
Trientine	Chelator and induces copper excretion	Gastritis, aplastic anemia, sideroblastic anemia	Initial: Adult 750 mg-1500 mg BID—QID Children—20 mg/kg/d BID—QID Maintenance 15 mg/kg/day	24 h urinary copper Nonceruloplasmin bound copper
Zinc	Stimulates metallothionein in intestine and liver and, inhibits intestinal absorption of copper	Gastritis, biochemical pancreatitis, doubtful immunosuppressant effects	Adults (>50 kg): 150 mg/day of elemental iron in three divided doses Children (<50 kg): 75 mg/day of elemental iron in three divided doses	24 h urinary copper Nonceruloplasmin bound copper 24 h urine zinc level
Tetrathiomolybdate	Tripartite complexes with protein and copper and renders it unabsorbable	Anemia, neutropenia, rarely hepatotoxicity	Still an experimental therapy and not commercially available. 20 mg 3× daily with meals, 20 mg 3× daily between meals