ABSTRACT
<p><b>OBJECTIVE</b>To evaluate the efficacy and safety of radix astragali and its compound prescription for treatment of β-thalassemia in children.</p><p><b>METHODS</b>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.</p><p><b>RESULTS</b>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 effects 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.</p><p><b>CONCLUSIONS</b>Therapy with radix astragali or its compound prescription is effective and safe in children with β-thalassemia.</p>