二巯丁二酸与青霉胺长期维持治疗肝豆状核变性疗效比较

Comparison of long-term therapeutic effects between succimer and penicillamine in hepatolenticular degeneration

目的 :比较二巯丁二酸与青霉胺长期维持治疗肝豆状核变性 (HLD)的疗效。方法 :将 12 0例HLD分 2组。A组 6 0例用二巯丁二酸 750mg ,po ,bid ;B组 6 0例用青霉胺 2 50mg ,po ,qid ;维持治疗时间 6mo～ 3a ,平均 1.5a±s1.0a。分 6mo～ 2a和 >2a以改良Goldstein分级法判断疗效。结果 :A组 2个治疗时间段的总有效率分别为 80 %和 85% ,B组为 58%和 59% ,差异均有显著意义(P <0 .0 5) ;A ,B 2组无效病人互换药物治疗后有效率分别为 2 7%和 6 8% ;A ,B 2组不良反应发生率分别为 15%和 37% ,差异均有显著意义 (P <0 .0 5)。结论 :二巯丁二酸长期疗效优于青霉胺 。

AIM: To compare the long term efficacy of succimer ( Suc ) in treating hepatolenticular degeneration ( HLD ) with that of penicillamine ( Pen ). METHODS: One hundred and twenty patients with HLD were divided into 2 groups: ( A ) 60 patients ( M 34, F 26; age 20 a± s 4 a) received Suc 750 mg, po . bid. ( B ) 60 patients ( M 31, F 29; age 18 a±5 a ) received Pen 250 mg, po , qid. The period of maintenance treatment varied from 6 mo to 3 a. average period was 1.5 a±1.0 a. Symptoms and therapeutic effects were evaluated by modified Goldstein scale. RESULTS: Observed in different period (6 mo～2 a and > 2 a) of treatment, the total effective rates of group A were 80 % and 85 % respectively, higher than those in group B (58 % and 59 % respectively ) ( P <0.05 ). The adverse reactions of group A were much less than those of group B ( P <0.05 ). Suc and Pen could increase urinary copper excretion effectively and continually. CONCLUTIONS: Suc is more effective and safer than Pen in the long lerm therapy of HLD.