羟基脲治疗重型β地中海贫血

Clinical study of hydroxyurea for the treatment of severe beta-thalassaemia

目的观察羟基脲治疗重症p一地中海贫血患儿的疗效并且对其不良反应进行评估。方法对15例重症B地中海贫血患儿应用羟基脲[10～20mg／（kg·d]治疗，治疗期间对患儿全血细胞计数、血红蛋白F、肝肾功能进行监测，治疗后6个月对疗效进行评估。结果治疗后5个月患儿血红蛋白及血红蛋白F较治疗前明显升高（72．5±6．1）vs（93．6±11．9），（45．58±15．79）vs（73．37±23．89），P〈0．05；而网织红细胞值明显降低（32．17±10．74）VS（15．87±7．29），P〈0．05。治疗期间未见明显的不良反应。结论羟基脲可改善重症β-地中海贫血患儿的贫血状态且无明显的不良反应。

Objective To investigate the use of hydroxyurea （HU） for the treatment of severe betathalassaemia （β-thal） children and to evaluate therapy-related adverse effects. Methods We examined the haematological effects of HU administered orally at the dosage of 10-20 mg/（ kg·d） in 15 severe β-thal children. Complete blood count, foetal haemoglobin （HbF）, liver enzymes were assessed before and after HU therapy. Response to therapy was evaluated at 6 months after treatment. Results The mean values of Hb and HbF were significantly higher at 5 months after HU treatment as compared with those before treatment [（72.5 ± 6.1） vs （93.6±11.9）] ;（45.58±15.79） % vs （73.37 ±23.89） %,P〈0.05]. In contrast to values before treatment, the mean reticulocyte count after therapy decreased significantly [ （32.17 ± 10.74 vs 15.87± 7.29）, P 〈 0.05 ]. No significant adverse effect was observed in our patients. Conclusion These restilts suggest that Hb levels in β-thal children can be improved by HU therapy in absence of obvious side effects.