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青霉胺与二巯丙磺钠反复交叉联合螯合治疗肝豆状核变性的临床疗效及安全性

Clinical efficacy and safety of chelation treatment with typical penicillamine in cross combination with DMPS repeatedly for Wilson's disease.

作者信息

Xu San-Qing, Li Xu-Fang, Zhu Hui-Yun, Liu Yan, Fang Feng, Chen Ling

机构信息

Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430030, China.

Department of Pediatrics, Guangzhou Women and Children's Medical Center, Guangzhou, 510120, China.

出版信息

J Huazhong Univ Sci Technolog Med Sci. 2013 Oct;33(5):743-747. doi: 10.1007/s11596-013-1190-z. Epub 2013 Oct 20.

Abstract

The aim of this study was to assess the clinical efficacy and safety of chelation treatment with penicillamine (PCA) in cross combination with sodium 2, 3-dimercapto-1-propane sulfonate (DMPS) repeatedly in patients with Wilson's disease (WD). Thirty-five patients with WD were enrolled. They were administrated intravenous DMPS in cross combination with oral PCA alternately which was practiced repeatedly, all with Zinc in the meantime. During the treatment, clinical observations and 24-h urine copper excretion as well as adverse effects of medicines were recorded and analyzed. Although the incidence of adverse effects was not significantly different after either intravenous DMPS or oral PCA treatment, levels of 24-h urine copper tended to be higher after short-term intravenous DMPS than that of oral PCA. Adverse effects in the course of intravenous DMPS were mainly neutropenia, thrombocytopenia, allergic reaction and bleeding tendency. As compared with oral PCA alone or intravenous DMPS alone, such repeated cross combination treatment could as much as possible avoid continued drug adverse effects or poor curative effect and had less chance to stop treatment in WD patients. Improved or recovered liver function in 71% of the patients, alleviated neurologic symptoms in 50% of the patients, and disappeared hematuria in 70% of the patients could be observed during the follow-up period of 6 months to 5 years after such combined chelation regimen. Chelation treatment repeatedly with oral penicillamine in cross combination with intravenous DMPS alternately could be more beneficial for WD patients to relieve symptoms, avoid continued drug adverse effects and maintain lifelong therapy.

摘要

本研究旨在评估青霉胺(PCA)与二巯基丙磺酸钠(DMPS)反复交叉联合螯合治疗肝豆状核变性(WD)患者的临床疗效及安全性。纳入35例WD患者,交替给予静脉注射DMPS与口服PCA反复交叉联合治疗,同时均服用锌剂。治疗期间,记录并分析临床观察指标、24小时尿铜排泄量及药物不良反应。虽然静脉注射DMPS或口服PCA治疗后不良反应发生率无显著差异,但短期静脉注射DMPS后24小时尿铜水平高于口服PCA。静脉注射DMPS过程中的不良反应主要有中性粒细胞减少、血小板减少、过敏反应及出血倾向。与单独口服PCA或单独静脉注射DMPS相比,这种反复交叉联合治疗可尽可能避免持续的药物不良反应或疗效不佳,WD患者停药机会更少。在联合螯合治疗方案后的6个月至5年随访期内,可观察到71%的患者肝功能改善或恢复,50%的患者神经症状缓解,70%的患者血尿消失。口服青霉胺与静脉注射DMPS反复交叉联合螯合治疗对WD患者缓解症状、避免持续药物不良反应及维持终身治疗可能更有益。

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