Wilson Disease Medication

Updated: Oct 18, 2016
  • Author: Richard K Gilroy, MBBS, FRACP; Chief Editor: Praveen K Roy, MD, AGAF  more...
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Medication

Medication Summary

The mainstay of therapy for Wilson disease is the use of chelating agents and medications that block copper absorption from the gastrointestinal (GI) tract.

Zinc and penicillamine are lifelong medications for patients with Wilson disease. Dosages vary with the severity of the disorder. Another chelating agent is trientine, which may be more easily tolerated than penicillamine. [19] Patients who do not respond to zinc therapy and who have increased activities of liver enzymes should be identified so that chelating agents may be added to the therapeutic regimen. [20, 21]

Other medications used to treat Wilson disease include anticholinergics, baclofen, gamma-aminobutyric acid (GABA) antagonists, and levodopa, to treat parkinsonism and dystonia symptoms; antiepileptics to treat seizures; and neuroleptics to treat psychiatric symptoms. In addition, protein restriction, lactulose, or both are used to treat hepatic encephalopathy.

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Chelators

Class Summary

Chelating agents bind excess copper. Ammonium tetrathiomolybdate is an investigational chelating drug used at the University of Michigan as an initial treatment for patients who present with neurologic or psychiatric manifestations. This drug works as a chelating agent and as an inhibitor of copper absorption from the GI tract. [22]

Penicillamine (Cuprimine, Depen)

Penicillamine forms soluble complexes with metals excreted in urine. It was the drug of choice before newer regimens were available. Because of extensive toxicities, alternative agents are used. It must be administered with pyridoxine 25 mg by mouth daily.

Trientine (Syprine)

Trientine is an effective oral chelator used to induce cupruresis. It is useful for patients who cannot tolerate penicillamine. It is indicated in Wilson disease if the initial presentation is hepatic. It should be administered with zinc.

Dimercaprol (BAL in Oil)

Dimercaprol is for refractory cases of Wilson disease that are not responding to first- or second-line chelation treatment.

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Nutrients

Class Summary

Nutrients are essential to normal growth and development, and they play a role in many metabolic processes.

Zinc (Galzin)

Zinc is a cofactor for more than 70 types of enzymes. It is approved for patients initially treated with a chelating agent. It should be used for maintenance after initial chelation therapy. Zinc acetate is the drug of choice in presymptomatic, pregnant, pediatric populations, and in some instance for maintenance in compliant patients who have undergone copper chelation therapy. It is a second-line therapy in patients with neurologic manifestations who do not tolerate chelation as a consequence of deterioration on this therapy.

Pyridoxine (Aminoxin, Pyri-500)

Pyridoxine is involved in synthesis of GABA within the CNS.

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