Deferasirox对铁质积聚有多大效果呢？

It was the first oral iron chelator approved by the FDA for routine use in 2005 and is now available in more than 80 countries. The oral suspension and dispersible tablets marketed in the United States are approved for the treatment of chronic iron overload caused by blood transfusions in adult and pediatric patients 2 years and older, and for the treatment of chronic iron overload in patients 10 years and older with non-transfusion-dependent thalassemia (NTDT) syndrome.

So, how effective is Deferasirox against iron buildup?

Therapeutic benefits of Deferasirox for treating iron accumulation:

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

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 to investigate the changes in serum ferritin (SF) and adverse reactions. 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 intermittently (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, P<0.01): 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, P < 0.05): 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, P<0.01), and there was no statistically significant difference in the mean cardiac MRI T2 values between the two groups.

Conclusion: It can significantly reduce SF levels in children with β-TM and show dose-dependent changes; it does not show obvious advantages in reducing cardiac iron load, but has a significant effect in reducing liver iron load. The adverse reactions of Deferasirox treatment are mainly increased liver enzymes and non-progressive increase in serum muscle NF.