补肾益髓法治疗β-地中海贫血92例临床研究  

Clinical Study on the Method for Tonifying Kideny and Supplementing Marrow for Treatment of 92 Cases of β-Miditerranean Anemia

作　　者：吴志奎[1] 张新华[2] 柴立民[1] 李敏[1] 蔡辉国[1] 王蕾[3] 方素萍[1] 陈玉英[1] 吕鑫霞[1] 黄有文[2] 王荣新[2] 陈佩珍[3] 

机构地区：[1]中国中医研究院广安门医院分子生物学研究室,北京市宣武区北线阁5号100053 [2]中国人民解放军第三○三医院 [3]中国医学科学院血液学研究所

出　　处：《中医杂志》2005年第4期277-279,共3页Journal of Traditional Chinese Medicine

基　　金：国家自然科学基金(30171199);广西自然科学基金(014402C);国家自然科学基金重大研究计划(90409003)

摘　　要：目的:研究中药益髓生血颗粒治疗β-地中海贫血(β-地贫)的临床效果及其疗效特点的分子机制。方法:用补肾益髓法代表方益髓生血颗粒在广西高发区治疗β-地贫92例,疗程3个月,观察治疗前后临床症状,血红蛋白(Hb)、抗碱血红蛋白(HbF)、红细胞(RBC)、网织红细胞(Ret)等血液参数变化及停药后随访观察,采用 PAGE、PCR-SSCP、RT-PCR、DNA 序列分析、mRNA 基因表达、DDRT-PCR 等技术研究疗效的分子机制。结果:经3个月治疗后,患者临床症状明显改善,Hb、HbF、RBC、Ret 等血液指标明显提高,停药后疗效平均维持3～6个月,92例中85例有效,有效率92.39%;中药治疗对各类基因型患者均有一定疗效,可明显提高血红蛋白珠蛋白 mRNA 表达和造血刺激因子 GM-CSFmRNA 表达,诱导 HbF 合成增加;降低铁蛋白基因表达,减少体内铁的蓄积。结论:益髓生血颗粒治疗β-地中海贫血效果显著;中药治疗不改变患者基因突变型,可明显调节功能基因表达。疗效特点为多靶点的整体效应。Objective:To study clinical effect and molecular mechanisms of Yisui Shengxue Granules for treatment of β-Miditerranean anemia.Methods:92 cases of β-Miditerranean anemia in high morbidity regions of Guangxi were treated with Yisui Shengxue Granules,a representative recipe of the method for tonifying kideny and supplementing marrow,for 3 months.Clinical symptoms and signs,hemoglobin(Hb),alkali-resistant hemoglobin(HbF),red blood cell(RBC),reticulocytes(Ret)etc.before and after treatment were observed and followed up.The molecular mechanisms were studied with PAGE,PCR-SSCP,RT-PCR,DNA sequential analysis,mRNA gene expression,DDRT-PCR and so on.Results:After treatment for 3 months,the symptoms improved significantly,Hb,HbF,RBC,Ret increased significantly,and the therapeutic effects lasted for 3～6 months after ceasing the treatment.Of the 92 cases,85 cases were effective,the effective rate being 92.39%.The medical treatment had a certain therapeutic effect for the patients of various gene types,and could significantly increase hemoglobin and globin mRNA and GM- CSFmRNA expressions,increase synthesis of HbF,decrease iron protein gene expression and accumulation of iron in the body. Conclusion:Yisui Shengxue Granules has a significant effect on β-Miditerranean anemia,and it does not change gene mutation type of patients and can significantly regulate expression of functional genes,with the characteristic of whole effect of multiple targets.

关 键 词：Β-地中海贫血 补肾益髓法 临床研究 益髓生血颗粒 血红蛋白(Hb) mRNA表达 DNA序列分析 抗碱血红蛋白 造血刺激因子 蛋白基因表达 分子机制 疗效特点 临床症状 中药治疗 网织红细胞 基因突变型 PCR 临床效果 治疗前后 随访观察 

分 类 号：R259[医药卫生—中西医结合]