Evaluation of therapeutic effects of standard treatment on children with transfusion-dependent thalassemia accompanied by iron overload
Objective To explore the therapeutic effect of standard treatment(regular blood transfusion and iron removal)on children with transfusion-dependent thalassemia(TDT)accompanied by iron overload.Methods A total of 62 children with TDT who received the first-time oral administration of deferasirox(DFX)dispersible tablets for iron removal treatment were selected.General clinical data of the children with TDT before treatment was collected,including age,gender,height,body mass,time of first blood transfusion,hemoglobin(Hb),and serum ferritin(SF)before transfusion,etc.The body height and body mass of the children were examined to assess the growth status once a year.Fasting venous blood was collected every three months to detect blood biochemical indicators such as Hb,serum ferritin,alanine aminotransferase(ALT),aspartate aminotransferase(AST)and creatinine,etc.Organ damage(elevated liver enzymes and creatinine,nausea,rash,joint pain,etc.)was recorded.The children were given with standard treatment and followed up for 2 years.Results 96.7％of the children with TDT had growth retardation before standard treatment.The children with TDT who exhibited growth retardation had lower Hb level before transfusion than those with normal growth and development had,while SF level was higher than that in children with normal growth and development(P＜0.05 or P＜0.01).SF of the children who received regular oral DFX for iron removal treatment for 2 years showed a decreasing trend yearly,and SF was decreased after taking the medication when compared to before enrollment(P＜0.05).There was no correlation between Hb level before blood transfusion and organ damage(P＞0.05),while the degree of iron overload was correlated with organ damage(P＜0.05).There was no new organ damage observed in the children with TDT who were followed up for 2 years with standardized treatment.There was no statistically significant difference in the incidence of organ damage in the children with TDT between before and after treatment(P＞0.05).Conclusion Growth retardation is common in children with TDT.High-volume blood transfusion and low iron load can improve growth retardation.DFX can effectively reduce iron load with less adverse reactions.
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