European-wide study confirms benefits of D-penicillamine and trientine for Wilson disease

Mar 31, 2011

Results from the first ever European-wide retrospective analysis presented today at the International Liver CongressTM have shown both D-penicillamine and trientine continue to be effective treatments, providing positive survival rates in patients with Wilson disease free from a liver transplant.

Wilson disease is a rare genetic storage disorder in which copper is not excreted by the body effectively, leading to excess copper build up, liver failure and damage to the brain (neurological problems). Worldwide the condition, affects approximately one in 30,000 people. If not treated, Wilson disease can be chronically debilitating and life threatening. While current guidelines recommend the use of chelating agents (D-penicillamine, trientine) as first line therapy of symptomatic patients, optimal treatment regimens are yet to be established.

In this retrospective multicentre cohort study the long-term outcomes of treatment with D-penicillamine and trientine were reviewed in 347 patients with the condition. Changes of medication were common in both groups and resulted in a total of 467 analysed treatments (D-penicillamine, n=326, trientine n=141). Following an average follow-up of 16.5 years, both treatments provided a similar and excellent survival rate and prevented disease progression with the need for in over 98% of patients.

Daniele Prati, EASL's Scientific Committee Member and Press Committee Chairman commented: "Not only does this study show that both treatments are effective options for patients with this rare and often debilitating condition, but it is also encouraging to see how European countries can work together and exchange data which is extremely valuable to guide best practice and evaluate existing treatment regimens. Research on rare liver diseases presents remarkable interest."

"To date, a very limited number of drugs for these conditions are marketed, leaving the majority of rare diseases without any effective treatment. Research into rare diseases and possible cures needs to be strongly encouraged."

Follow-up at 48 months showed that liver deterioration occurred in only 8 out of 515 treatments (D-penicillamine n=4, trientine n=4) while neurological deterioration was less frequent in the D-penicillamine group (7/326 treatments) compared to trientine (12/141, p≤=0.05). Improvements in liver function of symptomatic patients was similar between groups when data were stratified between first and second line treatments, with improvement rates during first line therapy in symptomatic patients of 90.7% for D-penicillamine and 92.6% for trientine.

Explore further: Philippines confirms second MERS case (Update)

More information: References

1. Weiss H.K et al, Efficacy and Safety of D-penicillamine and Trientine for the Treatment of Wilson Disease. Presented at the International Liver Congress 2011

2. Wilson's disease for patients and families, EuroWilson eurowilson.org/en/living/guide/what/index.phtml . Accessed March 2011.

3. Wilson Disease Association. www.wilsondisease.org/about-wilsondisease.php . Accessed March 2011

Related Stories

Recommended for you

Philippines confirms second MERS case (Update)

7 hours ago

A foreigner who flew to the Philippines from the Middle East has become the second confirmed case of MERS in the country, the health department said Monday, as a deadly outbreak in South Korea spreads alarm across Asia.

Immigrant children given adult dose of hepatitis A vaccine

Jul 04, 2015

About 250 immigrant children were given an adult dose of a hepatitis A vaccine at a Texas detention facility where they were being held with their mothers, according to U.S. Immigration and Customs Enforcement officials.

User comments : 0

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.