Subject headings Gandou decoction; dimercaptosuccinic acid; hepatolenticular

    degeneration; trace elements

    Abstract

    AIM To investigate the curative effect of integrated traditional Chinese and western

    medicine (TCM-WM) therapy in hepatolenticular dergeneration (HLD).

    

    METHODS Eighty patients with HLD were divided randomly into two groups

    (TCM-WM group and WM group), TCM-WM group (40 cases) were given

    oral dimercaptosuccinic acid (DMSA) 50 mg/kg body weight, twice daily and

    Gandou decoction once a day consecutively for 30 days. The efficacy was

    compared with that of 40 cases treated only with DMSA as the control. The

    changes of urinary trace and macro-elements were observed before and after treatment.

    

    RESULTS The total effectiveness rate of TCM-WM group was significantly

    higher than that of WM group (P<0.05), especially more patients with hepatic

    type responded well to treatment with TCM-WM than with only chelating agent

    DMSA. Both TCM-WM and WM therapy could obviously increase urinary

    trace and macro-element excretion (P<0.01, respectively).

    

    CONCLUSION TCM-WM therapy appears extremely promising as new

    cupruretic method for treatment of HLD. It is particularly suitable for the patients

    with hepatic type.

    INTRODUCTIONH epatolenticular degeneration (HLD) is an autosomal

    recessive disease of copper transport, resulting in copper accumulation and

    toxicity to the liver and brain affecting most commonly children or young adults

    and running an invariably fatal course if not adequately treated by decoppering

    therapy. At present, the gene (WD) has been mapped to chromosome 13 q 14.3^[1,2] ,

    both increasing awareness of the etiological mechanism of the illness and the

    introduction of new pharmacologic approaches to its treatment have demonstrated

    that this disease is certainly preventable and to some extent is curable. This study

    was designed to compare clinical therapeutic effects of TCM-WM and WM for HLD.

    MATERIALS AND METHODS

    Subjects

    Eighty patients with HLD were chosen for this study. They were diagnosed in

    accordance with the criteria proposed by Houwen^[3] and were randomly

    divided into integrated traditional and western medicine therapy (TCM-WM

    group) and western medicine therapy (WM group). Based on clinical symptoms,

    they were classified as neurological type (including Wilson type and pseudosclerosis

    type) and hepatic type^[4] . The severity of the disease was graded from Ⅰ to Ⅳ

    according to the modified Goldstein method^[5] . These two groups were comparable

    for their age, sex, course of disease, clinical classification and severity (Table 1).

    

    Table 1 Characteristics of patients of TCM-WM group and WM group