大剂量硫酸锌与小剂量青霉胺联合治疗儿童肝豆状核变性长期随访  

Long Term Follow up of Combined Therapy with Large dose Zinc Sulfate and Low Dose Penicillamine in Children with Hepatolenticular Degeneration

作　　者：李堂[1] 林荣军[1] 杜嗣廉[1] 曲政海[1] 

机构地区：[1]青岛医学院附属医院小儿内科

出　　处：《中华医学遗传学杂志》1999年第1期19-21,共3页Chinese Journal of Medical Genetics

摘　　要：目的总结大剂量硫酸锌与小剂量青霉胺联合治疗儿童肝豆状核变性（ｈｅｐａｔｏｌｅｎｔｉｃｕｌａｒｄｅ－ｇｅｎｅｒａｔｉｏｎ，ＨＬＤ）的远期效果。方法有症状患儿初期用小剂量青霉胺（每天８～１０ｍｇ／ｋｇ），加大剂量硫酸锌（＜６岁１００～１５０ｍｇ，６～８岁１５０～２００ｍｇ，９～１０岁２００～３００ｍｇ，＞１０岁３００ｍｇ，每天３次）联合治疗；病情好转后和症状前期患儿单用硫酸锌维持治疗。对３１例患儿进行４～１１年随访。结果３例症状前期患儿未出现异常；２８例有症状患儿中２３例（８２％）临床症状基本消失或明显好转，２例（７％）无改善，３例（１１％）死亡。全部患儿血铜持续低于正常；２４小时尿铜于服药后６个月均较治疗前降低，降至正常者５例（１６％），服药后１～２年降至正常者２６例（８４％）。疗程中２０例（６５％）有１次或多次血锌增高，２５例（８１％）２４小时尿锌增高。结论大剂量硫酸锌与小剂量青霉胺联合应用治疗儿童ＨＬＤ效果可靠，价格低廉，副作用小。Objective To summarize the long term effect of combined treatment with large dose zinc sulfate and low dose penicillamine in children with hepatolenticular degeneration (HLD).Methods The patients who had symptoms were treated with large dose zinc sulfate (100 150mg,<6yr;150 200mg,6 8yr;200 300mg,9 10yr;300mg,>10yr; 3 times a day) in addition to low dose penicillamine(8 10mg/kg/d) at the beginning of treatment. Zinc sulfate alone was given to the presymptomatic patients and it was used as maintenance therapy when clinical improvement was obtained. 31 children were followed up for 4 11 years.Results In 3 presymptomatic patients, no clinical abnormalities were found. Among 28 patients with symptoms, 23 patients (82%) had their symptoms and signs subsided or much improved, 2 patients(7%) remained unchanged, and 3(11%) died. Blood concentrations of copper were persistently lower than normal. Urine copper excretion of 24 hours was significantly lower than that before the combined therapy in all patients, and it became normal in 5 cases (16%) after 6 months of treatment, and in 26 cases (84%) after 1 2 years of treatment. Higher blood concentrations of zinc were found in 20 cases (65%), and higher urine zinc excretion was noted in 25 cases(81%) once or more times during the therapy.Conclusion Combined therapy of large dose zinc sulfate and low dose penicillamine is an effective, safe and cheap treatment for children with HLD.

关 键 词：肝豆状核变性 硫酸锌 青霉素 儿童 药物疗法 

分 类 号：R742.405[医药卫生—神经病学与精神病学]