Successful treatment of acute liver failure due to Wilson's disease: Serendipity or fortuity?

Background: Acute liver failure (ALF) may be the first and most dramatic presentation of Wilson's disease (WD). ALF due to WD (WD-ALF) is difficult to distinguish from other causes of liver disease and is a clear indication for liver transplantation. There is no firm recommendation on specific and supportive medical treatment for this condition.

Aim: To critically evaluate the diagnostic and therapeutic management of WD-ALF patients in order to improve their survival with native liver.

Methods: A retrospective analysis of patients with WD-ALF was conducted in two pediatric liver units from 2018 to 2023.

Results: During the study period, 16 children (9 males) received a diagnosis of WD and 2 of them presented with ALF. The first was successfully treated with an unconventional combination of low doses of D-penicillamine and zinc plus steroids, and survived without liver transplant. The second, exclusively treated with supportive therapy, needed a hepatotransplant to overcome ALF.

Conclusion: Successful treatment of 1 WD-ALF patient with low-dose D-penicillamine and zinc plus steroids may provide new perspectives for management of this condition, which is currently only treated with liver transplantation.