黄芪及其复方中药治疗小儿β地中海贫血的前瞻性临床研究

Prospective clinical study of radix astragali and its compound prescription for treatment of β-thalassemia in children

摘要:

目的：评价黄芪及其复方中药颗粒治疗小儿β地中海贫血的疗效及安全性。方法：采用随机对照双盲的临床试验法，57例入选患儿先按中间型及重型分层，再随机分入黄芪组、复方组(黄芪+党参+龟板)、安慰剂对照组，疗程12周，观察各组血液学指标及安全性指标。结果：经12周的治疗，中间型患儿复方组血红蛋白提升值为1.21±1.12 g/dL，黄芪组为1.05±0.80 g/dL，而对照组则下降了0.28±0.51 g/dL，3组比较差异有统计学意义（P<0.01）。疗程结束后复方组及黄芪组的Hb水平均显著高于对照组（P<0.05）。黄芪及其复方治疗均显著提升了胎儿血红蛋白水平，一定程度上提升了红细胞总数、平均血红蛋白含量及网织红细胞。中间型患儿复方组总有效率为64%，黄芪组为62%，均显著高于对照组（9%；P<0.01）。在重型患儿中也有类似以上效果，但逊于中间型患儿。服用药物对患儿白细胞总数、中性粒细胞总数、血小板总数及肝肾功能均无显著性影响。结论：黄芪及其复方治疗小儿β地中海贫血具有一定疗效且较安全。

Abstract:

OBJECTIVE: To evaluate the efficacy and safety of radix astragali and its compound prescription for treatment of β-thalassemia in children. METHODS: This study was a randomized, controlled, double-blind clinical trial. Fifty-seven children with β-thalassemia were randomly assigned to radix astragali, compound prescription (radix astragali+ codonopsis pilosula + tortoise plastron) and placebo control groups after stratifying the patients according to disease type (intermedia and major). The parameters of hematology and safety were assessed after 12 weeks of treatment. RESULTS: After 12 weeks of treatment, the mean Hb elevation levels in children with β-thalassemia intermedia from the compound prescription and the radix astragali groups were 1.21±1.12 and 1.05±0.80 g/dL respectively compared with -(0.28±0.51) g/dL in the placebo control group (P<0.01). Mean Hb levels in the compound prescription and radix astragali groups were significantly higher than in the placebo control group (P<0.05). Therapy with both radix astragali and its compound prescription increased fetal hemoglobin, red blood cell, mean corpuscular hemoglobin and reticulocyte levels in children with β-thalassemia intermedia. The total effective rates were 64% and 62% in children with β-thalassemia intermedia from the compound prescription and radix astragali groups respectively, which was significantly higher than in the placebo control group (9%; P<0.01). Therapy with radix astragali or its compound prescription in children with β-thalassemia major had similar but less favourable effets than the same therapy in children with β-thalassemia intermedia. White blood cell, neutrophil, platelet and hepatic and renal functions were not adversely affected by the medicines. CONCLUSIONS: Therapy with radix astragali or its compound prescription is effective and safe in children with β-thalassemia.