A recent retrospective analysis on 405 patients with Wilson disease analyzed the efficacy and safety of oral chelators for Wilson disease (Clin Gastroenterol Hepatol 2013; 11: 1028-35).
The authors noted that there were frequent changes in medication; in total, 471 monotherapies were analyzed: 326 patients with D-penicillamine (DPA) and 141 with trientine. Trientine was a first line treatment in only 38 patients. About 50% of patients presented with hepatic symptoms, about 20% neurologic symptoms, about 15% with combined hepatic/neurologic symptoms and the remainder, ~10%, were asymptomatic.
- 9 of 326 with DPA and 3 of 141 with trientine underwent liver transplantation.
- Adverse effects were more common with DPA, including arthralgias (8.9%), proteinuria (6.1%), ANA antibodies (6.7%), gastric complaints (2.5%) and polyneuropathy (1.8%). 28.8% of DPA stopped therapy due to adverse effects.
- Adverse effects with trientine resulted in stopping treatment in 7.1% and included arthralgias in 2.8%, and gastric complaints in 1.4%.
- Hepatic improvements were observed in >90%
The authors summarize their findings:
“Both DPA and trientine were equally and highly effective in controlling liver disease…In light of recent reports of hepatic deterioration under zinc therapy, the current data emphasize the role of these chelating agents in the treatment of symptomatic hepatic patients.”
Related blog post:
Finding the Right Specialist | gutsandgrowth This post has link to AASLD guidelines for Wilson disease.