How is Wilson disease treated?

Updated: Feb 14, 2019
  • Author: Richard K Gilroy, MBBS, FRACP; Chief Editor: Praveen K Roy, MD, AGAF  more...
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Answer

The mainstay of therapy for Wilson disease is pharmacologic treatment with chelating agents such as D-penicillamine and trientine. [2] Other agents include sodium dimercaptosuccinate, dimercaptosuccinic acid, zinc, and tetrathiomolybdate. [13] Zinc salts act as inductors of methallothioneins, which favor a negative copper balance and a reduction of free plasmatic copper. [2]

The use of surgical decompression or transjugular intrahepatic shunting (TIPS) in the treatment of portal hypertension is reserved for individuals with recurrent or uncontrolled variceal bleeding that is unresponsive to standard conservative measures.

After the initiation of therapy with a chelating agent, the patient needs to be aware of potential adverse effects of the agents with which he or she is being treated. For instance, some of the concerning adverse effects are those commonly associated with penicillamine use. In addition, a patient must also be aware of the potential to develop worsening of some symptoms when chelation is started; in particular, patients with neurologic signs and symptoms can see worsening of these with chelation, and, in some instances, therapy needs to be reduced or stopped. Laboratory tests in patients started on penicillamine should include hematology and biochemical monitoring, as well as urinalysis.

With clinical progression, acute liver failure, or worsening hepatic function, the patient must be evaluated at a center with expertise in Wilson disease and the capability to perform liver transplantation.

Orthotopic liver transplantation is curative treatment for Wilson disease. Also see Liver Transplantation.


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