Abstract: OBJECTIVE: To investigate the effect and safety of Gandouling plus low-dose D-penicillamine for treating patients with Wilson's disease(WD) who have neurological symptoms.METHODS: WD patients with neurological symptoms were divided into two groups: a treatment group(n = 53) and a control group(n = 50). The treatment group received anti-copper therapy with a combination of Gandouling and low-dose D-penicillamine(10 mg/kg), whereas the control group was with conventional dose D-penicillamine(20 mg/kg) monotherapy. The clinical efficacies, adverse reactions, and results of the various hematological and biochemical investigations were recorded and analyzed statistically.RESULTS: Overall, 98.11% of the WD patients treated with the combined therapy experienced alleviation of their neurological condition(paralleled by asignificantly improved Global Assessment Scale score or remained stable). Their white blood cell and platelet counts stabilized, and their liver function was improved or remained stable. The combined therapy also obviously promoted improved24-h urinary copper excretion. Only 15.09% of the WD patients with the combined therapy experienced adverse reactions, including neurological deterioration in one case(1.89%) and hepatic worsening in one case(1.89%), which was less frequent than that in the control group given conventional-dose D-penicillamine monotherapy.CONCLUSION: Treating WD patients with neurological symptoms using Gandouling plus low-dose D-penicillamine is effective and safe.

Key words: Hepatolenticular degeneration, Penicillaminate, Neurologic manifestations, Gandouling