人纤溶酶原治疗先天性纤溶酶原缺乏症的研究进展
Research progress of plasminogen in the treatment of congenital plasminogen deficiency
于丽杰 1王磊 1王婷 1张慧 1王俊龙 1王伟2
作者信息
- 1. 上海益临思医药开发有限公司,上海 200437
- 2. 吉林大学第一医院儿科门诊,吉林长春 130000
- 折叠
摘要
先天性纤溶酶原缺乏症(congenital plasminogen deficiency,C-PLGD)又称Ⅰ型纤溶酶原缺乏症(type Ⅰ plasminogen deficiency)、纤溶酶原缺乏症(plasminogen deficiency)或低纤溶酶原血症(hypoplasminogenemia),是一种超罕见的常染色体隐性遗传性纤溶系统疾病,主要表现为全身黏膜上富含纤维蛋白的木样假膜病变在血管外异常堆积、生长.若不及时治疗,这些病变可能会损害正常组织及器官功能,影响患者生活质量.目前,国内尚无获准用于治疗C-PLGD的特异性替代疗法.因此,治疗方法仅限于非特异性疗法及包括手术在内的干预措施,而所有这些方法均被证明效果不佳.国外临床研究表明使用人谷氨酸-纤溶酶原替代治疗可持续提高纤溶酶原活性,改善C-PLGD患者临床症状,且耐受性良好.现对人纤溶酶原治疗C-PLGD的研究进展进行概况综述,以期为其治疗纤溶酶原缺乏症的临床应用及研究提供一定的思路和依据.
Abstract
Congenital plasminogen deficiency(C-PLGD),also referred to as type Ⅰ plasminogen deficiency,plasminogen deficiency or hypoplasminogenemia,is an ultra-rare autosomal recessive disorder of the fibrinolytic system,which is mainly manifested by the abnormal extravascular accumulation and growth of fibrin-rich,ligneous pseudomembranous lesions on mucous membranes throughout the body.Left untreated,these lesions may impair normal tissue and organ function,and impact patients'quality of life.Currently,there is no specific replacement therapy approved for the treatment of C-PLGD in China.As such,treatment options have been limited to non-specific therapies and interventions,including surgery,all of which have been proved to be less effective.Foreign clinical trials have shown that replacement therapy with human glutamate-plasminogen can consistently increase plasminogen activity and improve the clinical symptoms of C-PLGD patients,and it is well tolerated.This article provided an overview of the research progress on the treatment of C-PLGD with human plasminogen,in order to provide some ideas and basis for its clinical application and research in the treatment of plasminogen deficiency.
关键词
先天性纤溶酶原缺乏症/木样假膜病变/谷氨酸-纤溶酶原Key words
congenital plasminogen deficiency/igneous pseudomembranous lesions/glutamate-plasminogen引用本文复制引用
出版年
2024
NETL
NSTL
万方数据