试用马利兰治疗β-地中海贫血  

Further Study on the Treatment of Severe β-Thalassemia with Myleran

作　　者：梁植权[1] 刘利娟[2] 杨克恭[1] 陈松森[1] 王荣新[2] 敖朝晖 刘德培[1] 贾佩臣[1] 查丹玉[2] 金和谦[2] 黄有文[2] 

机构地区：[1]中国医学科学院基础医学研究所 [2]中国人民解放军第303医院

出　　处：《中国医学科学院学报》1991年第2期133-136,共4页Acta Academiae Medicinae Sinicae

基　　金：国家自然科学基金

摘　　要：对3例重型β-地中海贫血(β-地贫)患儿试用马利兰治疗。患儿每日口服马利兰(0.2mg/kg),20d后,Hb,HbF,RBC等主要血液学指数均有所改善。临床症状缓解,肝脾肿大减轻。2例患儿姐妹染色体互换(SCE)有轻度增加,停药后即可恢复,1例基本无变化,提示所用剂量的马利兰对患者染色体DNA无明显损伤。用多聚酶链反应(PCR)和寡聚核苷酸探针杂交测定13例经马利兰治疗的重型β-地贫患儿的DNA,在22条β-地贫染色体中发现有7种β-珠蛋白基因突变类型,提示马利兰对不同突变类型β-地贫均有疗效。In a previous paper we reported the results of treatment of five patients with β-thalassemia major with myleran. Three more cases are reported in this paper.After 40-50 days of oral administration of myleran, major hematological findings (Hb, HbF, RBC) began to increase and remained at higher level for 4-5 months before dropping back to the original. The results of analysis of sister chromatid exchange (SCE) showed no irreversible damage on chromosomal DNA of the patients at the dosage used(0.2mg/kg · d).It was also demonstrated that the effect of treatment does not seem to be related to the types of β-globin gene mutations of the patients.

关 键 词：马利兰 地中海贫血 

分 类 号：R556.610.5[医药卫生—血液循环系统疾病]