地拉罗司治疗铁质积聚的效果怎样？

Thalassemia is a rare genetic disease. In addition to iron removal and blood transfusion, most patients in China use oral dispersible tablets as treatment.

Deferasirox is an iron chelator product developed by the Swiss pharmaceutical company Novartis. It is the first oral iron-depleting agent approved by the U.S. FDA for routine use. It is approved for use in patients ≥2 years old with chronic iron overload caused by blood transfusion. In Europe, it is recommended as a first-line drug for patients with thalassemia iron overload over 6 years old.

Deferasirox is a life-saving drug for patients with thalassemia. Although blood transfusion and iron elimination may alleviate the progression of the disease, the effect is not as good as oral deferasirox. The safety and convenience of oral deferasirox treatment are far better than blood transfusion, and the side effects are also smaller. 

So, how effective is deferasirox in treating iron buildup?

Objective: To study the efficacy and safety of deferasirox-desferrioxamine sequential therapy in the treatment of iron overload in β-thalassemia major (β-TM).

Methods: 150 children with β-TM who had blood transfusion and iron overload were randomly divided into 3 groups and treated with deferasirox (group I), deferasirox-deferoxamine sequential therapy (group II), and deferoxamine (group III) respectively. The clinical data and treatment compliance of the three groups of children were statistically analyzed. The observation period is 1 year.

Results: There was no difference in the total blood transfusion volume among the three groups. The SF before and after treatment was significantly lower than that of group I and group II. After treatment, the SF of group I and group II was significantly lower than that of group III (P<0.01).

The number of delayed bone age cases in groups Ⅰ and Ⅱ was significantly reduced before and after treatment, and the number of cases in group Ⅲ after treatment was significantly increased compared with groups Ⅰ and Ⅱ (P<0.01). The number of cases of abnormal liver function in Groups I and II after treatment were significantly reduced compared with those before treatment, and the number of cases of abnormal liver function in Group II after treatment was significantly reduced compared with Groups I and III (P<0.05). There were significantly more cases of poor compliance in group III than in groups I and II (P<0.01). After treatment, the height and weight growth in groups I and II were significantly higher than those in group III (P<0.01).

Conclusion: -Desferrioxamine sequential therapy is safe and effective in the treatment of β-TM iron overload and can be used as one of the options for the treatment of iron overload.