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Wilson's disease

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What is Wilson's disease?

Wilson's disease is a rare genetic disorder caused by the accumulation of copper throughout the body.

In the human body, copper plays an important role in the development of healthy bones, nerves, collagen and the skin pigment melanin. Copper is normally absorbed from food and excess copper is excreted through bile. Bile is produced by the liver and carries toxins and waste away from the body, through the digestive system.

In people with Wilson's disease, there is a defective enzyme that results in excess copper not being removed. Instead, it leaves the bloodstream and accumulates in various organs and structures including the liver, brain, spinal cord, eyes and kidneys. About 50% of people diagnosed with Wilson's disease have symptoms that are localized to just their liver.

Copper is toxic in large amounts. As tissues become damaged by the build-up of copper, the healthy tissue is replaced by scar tissue, which cannot perform the normal functions of the organ involved and the organ ceases to work properly.

Wilson's disease affects around one in every 30,000 people. It cannot be cured, but can be very well managed if diagnosed early. The copper accumulation starts from birth, but symptoms can take many years to become apparent. It is typically diagnosed in people between 5-35 years of age and affects men and women equally.

Causes

People have two copies of most genes and inherit one copy from each parent. A genetic disease occurs when one or both parents pass on a mutated gene to a child at conception. In Wilson's disease, a child inherits a copy of the genetic mutation from both their parents.

The gene responsible for Wilson's disease is called ATP7B. When this gene is mutated, excess copper cannot be moved from the liver effectively.

About one in 100 people are carriers of the ATP7B gene. Carriers of the gene do not become ill and do not need medical treatment. When both parents of a child carry it, there is a:

Signs and symptoms

The symptoms experienced by people with Wilson's disease may vary, depending on which organs are affected by the copper accumulation.

Although the condition is present at birth, the symptoms do not become apparent until the copper has accumulated in the organs. Some people diagnosed with Wilson's disease may not even have any symptoms; instead, their condition is discovered during routine physical examinations or laboratory tests.

Liver-related signs and symptoms

Liver symptoms are typically the first to develop. The toxic effects of the copper on the liver cells may cause:

Some people with Wilson's disease may not develop any symptoms until their condition has reached the stage of acute liver failure, which can develop very suddenly.

Central nervous system-related signs and symptoms

Symptoms that affect the central nervous system and mental health often appear once the liver has accumulated a lot of copper.

These symptoms are more often apparent in adults, but can occur in children and include:

Other signs and symptoms

Methods for diagnosis

Your doctor will ask about your medical history and symptoms and will perform a thorough physical examination.

Genetic testing

When there is an established family history of Wilson's disease, early diagnosis can prevent symptoms and potential organ damage. If a relative has previously been diagnosed with Wilson's disease, genetic testing may be an option, but as there are many mutations that can cause the condition, there is no single test that is appropriate for everyone.

Blood tests

Liver function and full blood counts

Blood tests are used to assess the function of the liver and to measure the levels of red blood cells, white blood cells and platelets. People with Wilson's disease will typically have an abnormal liver function test result and low blood count levels.

Copper levels

Since the copper is deposited into the organs and not circulating in the blood, most people with Wilson's disease actually have a lower-than-normal level of copper in their blood.

Ceruloplasmin levels

Ceruloplasmin is a protein that carries copper in the bloodstream. Most people with Wilson's disease have a lower-than-normal level of this protein in their blood.

Urine test

People with Wilson's disease have abnormally high levels of copper in their urine.

Eye examination

A doctor will use a microscope with a high-intensity light source to check eyes for Kayser-Fleischer rings. Wilson's disease is also associated with a specific type of cataract, called a sunflower cataract, which may also be apparent on examination.

Imaging

To exclude other possible conditions that may be causing neurological symptoms, diagnosis may include imaging scans of the head such as magnetic resonance imaging (MRI) or computerized tomography (CT).

Liver biopsy

To assess damage to the liver and measure the amount of copper that has accumulated, a small sample of liver tissue may be removed (biopsied) for further testing.

Types of treatment

The aim of treatment is to reduce the amount of copper in the body and to control any symptoms. Treatment is lifelong and delays in starting treatment can potentially cause irreversible damage.

Medications

Zinc acetate

Zinc increases the levels of an intestinal protein called metallothionein, which has a high affinity for copper. The metallothionein reduces the absorption of copper and increases its excretion. Zinc can cause an upset stomach for some people.

D-penicillamine and trientine

Copper chelation therapy prompts organs to release copper into the bloodstream, which is then filtered by the kidneys and released into urine.

D-penicillamine is often the primary medication for copper chelation, but it commonly causes side effects such as skin problems, bone marrow suppression and worsening of neurological symptoms.

Trientine works similarly to D-penicillamine, but has fewer associated side effects.

Vitamin B6

Vitamin B6 supplements may help ease nervous system symptoms.

Potassium

Potassium supplements can reduce absorption of dietary copper.

Dietary changes

People with Wilson's disease may be advised to follow a diet that is low in copper, which may include avoiding chocolate, dried beans, dried fruits, mushrooms, nuts, offal, peas, shellfish and wholewheat products. People with poor liver function may be advised to avoid excess alcohol consumption.

Liver transplantation

A liver transplantation may be considered when there is severe liver damage, acute liver failure, or when no other treatment options have proved effective.

Potential complications

Without medical treatment, complications of Wilson's disease can include:

Prognosis

Wilson's disease cannot be cured, but with proper management, a person with Wilson's disease can usually live a normal life. However, people who have advanced Wilson's disease, or rapidly progressive liver failure, can expect serious health consequences.

Prevention

Wilson's disease cannot be prevented, but those with a family history may benefit from being tested for the disease before symptoms appear, as an early diagnosis can reduce or prevent organ damage.

References

  1. Clearinghouse The NIDDK National Digestive Diseases Information. “Wilson Disease.” Text. Accessed October 8 2014. link here
  2. myVMC. “Wilson’s Disease. myVMC. Accessed October 8 2014. link here
  3. Wilson’s Disease | Health.” Patient.co.uk. Accessed October 8 2014. link here
  4. Wilson Disease” May 2 2014. link here
  5. Wilson Disease.” Cleveland Clinic. Accessed October 8 2014. link here
  6. Wilson’s Disease.” Accessed October 8 2014. link here
  7. Wilson’s Disease.” Better Health Channel. Accessed October 7 2014. link here
  8. Wilson’s Disease | myVMC.” Accessed October 8 2014. link here
  9. Wilson’s Disease - Health - Beliefnet.com.” Accessed October 8 2014. link here
  10. Wilson’s Disease - Health - Beliefnet.com.” Accessed October 8 2014. link here

9 Most frequently asked questions (FAQs)

What is Wilson's disease?
Wilson's disease is a rare genetic disorder that is associated with the accumulation of excess copper throughout the body.
What are the symptoms of Wilson's disease?
The symptoms of Wilson's disease can vary widely and depend on which organs or structures have been affected by the build-up of copper. When the liver is affected, some of the symptoms may include jaundice, nausea, loss of appetite, vomiting, weakness, and swelling (edema). When the central nervous system is affected, some of the symptoms may include: anxiety, depression, confusion, movement disorders, clumsiness, speech problems, and personality changes.
What causes Wilson's disease?
Wilson's disease is caused by a mutation on chromosome 13 in the gene ATP7B. Mutations in this gene can lead to the abnormal accumulation of copper throughout the body.
Who gets Wilson's disease?
Wilson's disease is something you are born with. If both of your parents carry the genetic mutation responsible for the condition, you have a one in four chance of having it. It is estimated to affect around one in every 30,000 people and affects men and women equally.
How is Wilson's disease diagnosed?
The diagnosis of Wilson's disease is typically based on: blood and urine tests that measure the levels of copper, ceruloplasmin, liver function and full blood count; eye examinations that identify the characteristic golden-brown discoloring caused by deposits of excess copper (Kayser-Flesicher rings), and; a liver biopsy (if needed).
How is Wilson's disease treated?
Treatment for Wilson's disease involves reducing the level of copper in the body and preventing its further accumulation. Treatment is lifelong and may include medication to eliminate excessive build-up of copper, medication to reduce further absorption of copper and following a low-copper diet. Severe cases of liver damage may require liver transplantation.
Can Wilson's disease be cured?
There is presently no known cure for Wilson's disease, but if diagnosed early, treatment allows for an excellent outcome.
Will Wilson's disease clear on its own?
Without medical treatment, Wilson's disease can prove fatal.
Can Wilson's disease be prevented?
There is no prevention for Wilson's disease.

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About this article

Title: Wilson's disease

Author: Dr Idan Ben-Barak PhD, MSc, BSc (Med)

First Published: 13 Jul 2015

Last reviewed: 17 Jan 2022

Category: Information on Wilson's disease

Average rating: 4.2 out of 5 (1556 votes)

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