Desirox治疗铁质积聚疗效怎么样？

Thalassemia (thalassemia for short) is a group of hereditary hemolytic anemias. As the most common single-gene genetic disease in humans, thalassemia is inherited with incomplete autosomal dominant inheritance. The treatment of thalassemia mainly relies on regular blood transfusion and iron removal therapy. However, multiple blood transfusions can easily lead to iron accumulation after massive destruction of red blood cells. Excess iron is deposited in important organs of the body, leading to complications such as heart failure, arrhythmia, and liver cirrhosis. Therefore, iron-removing treatment should be given at the same time. There are currently oral iron-removing drugs such as Desirox. How effective is Desirox in treating iron accumulation?

Therapeutic benefits of Desirox for treating iron buildup:

To compare the effects of Desirox and deferoxamine (DFO) in the treatment of transfusion-dependent thalassemia (TDT), and evaluate the advantages and limitations of monotherapy.

Methods: The case data of TDT combined with iron overload and treated with Desirox or DFO in the outpatient clinic from January 2013 to June 2015 were retrospectively analyzed.

Fifty-eight children in the Desirox group were treated with Desirox 20-35 mg/(kg·d), and 27 children in the DFO group were treated with DFO 25-45 mg/(kg·d), 5 days a week. The patients were followed up for 1 year since taking the medication, and serum ferritin (SF) level, liver and kidney function, and blood routine were measured every 3 to 6 months.

Results: There was no statistically significant difference in the median age, average iron intake rate and pre-treatment SF between the two groups of children. After 6 months of follow-up, the mean SF decreases in the Desirox group and DFO group were 168 (-2 650, 7 254) ng/mL and 170 (-260, 599) ng/mL respectively (P>0.05). The decrease in SF in the Desirox group was positively correlated with dose (P<0.05). After 7 to 12 months of follow-up, the mean SF decreases in the Desirox group and the DFO group were 212 (-370, 795) ng/mL and -1 330 (-2 454, -206) ng/mL respectively (P<0.05).

Conclusion: It has advantages over DFO in the treatment of TDT, and the maximum dose within the tolerance range should be used. Desirox can stabilize SF and should be used long-term. The safe dose of DFO is not effective in children with massive blood transfusions.