地拉罗司对铁质积聚能有多大的疗效呢？

It was developed by Novartis and is currently the only oral iron chelator. It was approved by the FDA in November 2005 for use in patients 2 years old and older with chronic iron overload caused by blood transfusion. So, how effective can deferasirox be in treating iron accumulation?

Therapeutic effects of deferasirox in treating iron accumulation:

To explore the efficacy and safety of iron chelator deferasirox in the treatment of iron overload in children with β-thalassemia major (β-TM).

Methods Twenty-four children with β-TM iron overload who received regular blood transfusions were randomly selected to participate in a clinical study of different doses of deferasirox, and the changes in serum ferritin (SF) and adverse reactions were investigated. The cardiac MRI T2 and liver MRI T2 values ​​of children who continued to take deferasirox for 5 years were compared with those of children who were treated with deferoxamine combined with deferiprone during the same period (control group).

Results: The initial dose of deferasirox of 20-30 mg/kg per day had no obvious effect on children with iron overload. After the dose was increased to 30-40 mg/kg per day, the SF level dropped significantly (U=58, P0.01). The most common adverse reaction was an increase in serum liver transaminases, followed by a non-progressive increase in serum creatinine.

The SF level of the 5-year continuous deferasirox treatment group was significantly lower than that of the control group (1748±481 ng/mL vs 3462±1744 ng/mL, P0.05); the liver MRI T2 value was significantly higher than that of the control group (8.5±2.9 ms vs 2.7±1.9 ms, P0.01). There was no statistically significant difference in the mean cardiac MRI T2 values ​​between the two groups.

Conclusion: Deferasirox can significantly reduce SF levels in children with β-TM, and shows dose-dependent changes; it does not show obvious advantages in reducing cardiac iron load, but has a significant effect in reducing liver iron load.

It is a tridentate iron chelator that combines with ferric iron ions to form a complex in a ratio of 2:1 and is excreted in the feces, thereby reducing iron storage in the body. Due to the continued presence of deferasirox in plasma, plasma non-transferrin-bound iron can be continuously reduced and the iron formed by toxicity in the body can be directly removed.