Should Liver Transplant Come Sooner in Wilson Disease?

Severe neurological symptoms, not liver failure, should be the signal to start thinking about a liver transplant in Wilson Disease, French researchers say.

Wilson Disease, an ailment in which toxic amounts of copper build up in patients, causing severe neurological problems, is easily treated in early stages.

Therapy aimed at halting its progression is done with chelating agents to remove the copper and diet restrictions to limit copper intake, such as by not eating chocolate, nuts, shellfish, organ meats or mushrooms.

If the copper continues to accumulate it will damage the liver.

In the late stage of the illness, liver transplantation is an option, but usually only when the liver has stopped functioning despite treatment. According to the Wilson Disease Association, only 5% of patients have liver transplants, and only when the liver has failed.

But some researchers believe transplantation should be considered earlier.

Reporting at the International Liver Congress in Barcelona, researchers from France said they had transplanted livers into Wilson Disease patients even though the patients did not have decompensated liver function.

Lead study author Rodolphe Sobesky, MD, of the Aphp-Hopital Paul-Brousse, Villejuif and colleagues reported on 15 patients, mean age 20.4 years who had liver transplants to cure their Wilson Disease.

All had severe neurological complications prior to the transplant.

Those included dystonic postures, tremor, chorea, and akineto hypertonic syndrome.

In 11 patients, there was a significant improvement in neurological symptoms after the transplant.

That also meant a significant improvement in quality of life.

Four patients had no improvement and died because of infectious complications after surviving a mean of 13 months.

The deceased patients had severe akineto hypertonic syndrome and one a rigid dystonia limiting movements and swallowing.

Despite the deaths, the researchers conclude that “Liver transplantation is an effective treatment option in Wilson Disease for patients with worsening neurological symptoms despite medical treatment, even in the absence of liver failure.”

They noted that having a long history of fixed neurological symptoms could be an indication that patients will not improve post-transplant.