Desirox治疗铁质积聚患者疗效如何？

Indications:

1. Patients with chronic iron overload transfusion hemosiderinosis secondary to blood transfusion who are 2 years old or older will develop symptoms of chronic iron overload when serum ferritin continues to be >1000 L. At this time, iron chelation therapy should be started.

2. Patients receiving ASCT can also be given iron chelator treatment when iron overload occurs;

3. It can be used to treat reversible renal insufficiency caused by Fanconi syndrome.

Desirox’s effectiveness in treating patients with iron accumulation:

To evaluate the efficacy and safety of Desirox, a new oral iron chelator, in the treatment of iron overload in patients with β-thalassemia major (β-thalassaemia).

Results: None of the 23 patients with severe β-thalassaemia used iron chelators regularly before treatment, and the iron overload condition was severe [the average baseline value of serum ferritin was (5433.96±2873.90) μg/L]. Desirox at 20 mg·kg-1·d-1 could maintain the patient's iron overload in a balanced state, and there was no significant change in serum ferritin levels before and after treatment; with the extension of treatment time, the dosage increased, ≥30 mg·kg-1·d-1 of Desirox can bring patients' iron overload to a negative balance state, and the difference in serum ferritin levels before and after treatment is statistically significant (P<0.01).

There were no serious adverse events of Desirox during the 3-year follow-up. The common adverse events were mild increases in liver transaminase and serum creatinine. All 23 patients were able to take the medicine on time and successfully completed the clinical study, with no deaths. Conclusion As a new type of oral iron chelator, Desirox can significantly improve the iron overload status of patients, and its efficacy is related to treatment time and drug dosage.

The simple medication method, good safety and tolerability can significantly improve the quality of life of patients and better meet the treatment needs of patients with iron overload.