羟基脲治疗中间型β地中海贫血的疗效观察

Curative Effects of Hydroxyurea on the Patients with β-thalassaemia Intermadia

目的:研究中间型β地中海贫血患者的临床特点,羟基脲对中间型β地中海贫血患者的疗效以及不良反应。方法:将29例患者分为羟基脲治疗组及未接受羟基脲治疗的对照组,观察和对比两组患者的疗效,对不良反应及输血情况进行评估。结果:羟基脲治疗3个月后血红蛋白水平即有上升,网织红细胞数在羟基脲治疗1年后有明显下降,而血清铁蛋白一直维持在较低水平。未接受羟基脲治疗组患者治疗后血红蛋白及网织红细胞水平均无明显改善,而血清铁蛋白水平逐渐升高。羟基脲组患者在羟基脲治疗1年后有12例患者脱离输血,治疗有效率为85.71%,而未接受羟基脲治疗组患者在治疗后输血依赖情况未改善。所有接受羟基脲治疗的患者,经过随访1年未发生严重不良反应。结论:羟基脲对中间型β-地中海贫血具有较好疗效,患者无严重不良反应,其长期疗效以及其长远不良反应还有待继续观察。

Objective: To investigate the clinical features of β-thalassaemia intermediate( TI) patients and the curative effect and side reactions of hydroxyurea therapys. Methods: Tw enty nine patients w ith TI w ere divided into hydroxyurea therapy group and no hydroxyurea therapy group; the curative effect and side reactions in 2 groups w ere compared; the situation of blood transfusion in the 2 groups w as evaluated. Results: In hydroxyurea therapy group,the hemoglobin level increased after treatment for 3 months; the reticulocyte percentage obviously decreased after treatment for 12 months; the serum ferritin had been maintained at a low level; w hile in no hydroxyurea therapy group,the levels of hemoglobin and reticulocytes w ere not significantly improved after treatment,the serum ferritin level gradually increased. In hydroxyurea therapy group,12 cases w ere out of blood transfusion after treatment for 12 months,effective rate of treatment w as 85. 71%; w hile in no hydroxyurea therapy group,the blood transfusion dependency w as not improved after treatment. No serious side reactions w ere found in all the hydroxyurea treated patients. Conclusion: The hydroxyurea show s a better curative effect on TI patients,no serious side reactions occur in all the patients treated w ith hydroxyurea,but the long-term curative effect and side reactions should be observed continuously.