Deferasirox对铁质积聚有多大的效果呢？

It is currently the only oral iron chelator developed by Novartis. It was approved by the FDA in November 2005 for use in patients 2 years and older with chronic iron overload caused by blood transfusions.

In December 2012, Deferasirox was approved by the European Commission for the treatment of chronic iron overload in patients aged 10 years and older with non-transfusion-dependent thalassemia (NTDT) syndrome who require chelation therapy due to contraindications or insufficiency of deferoxamine mesylate therapy. So, how effective is Deferasirox against iron buildup?

Therapeutic benefits of Deferasirox for 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).

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 in the 5-year continuous Deferasirox treatment group was significantly lower than that in 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 in the control group (8.5±2.9 ms vs 2.7±1.9 ms, P<0.01). 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 shows dose-dependent changes; it does not show obvious advantages in reducing iron load in the heart, but has a significant effect in reducing iron load in the liver. The main adverse reactions of Deferasirox treatment are increased liver enzymes and non-progressive increase in serum muscle NF.