恩瑞格治疗铁质积聚疗效如何？

(Deferasirox) 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, Enrig (deferasirox) was approved by the European Commission for the treatment of chronic iron overload in patients aged 10 years and above with non-transfusion-dependent thalassemia (NTDT) syndrome who require chelation therapy due to contraindications or insufficiency of deferoxamine mesylate therapy.

Enrig (deferasirox) is a drug commonly used to relieve iron accumulation. How effective is Enrig in treating iron accumulation?

The study screened and optimized the formulation of Enrig (deferasirox) dispersible tablets with disintegration time limit and 30 Minute cumulative dissolution was used as the evaluation index, and the dosage of diluent microcrystalline cellulose (MCC) and lactose, the mass ratio of disintegrant cross-linked povidone (PVPP), and the dosage of cosolvent sodium dodecyl sulfate (SDS) were used as factors. The L9(34) orthogonal test table was used to design the test plan, and the prescription of Enrig (deferasirox) dispersible tablets was screened and the prescription was verified. The results showed that the optimized prescription was 12.5% ​​MCC, 27.5% lactose, 18% PVPP (internally added), and 1.0% SDS. In the verification test, deferasirox dispersible tablets completely disintegrated within 120 s, with an average 30-minute cumulative dissolution of 86.0%, which has great advantages over ordinary tablets. It can be seen that the dissolution rate, disintegration time and other indicators of Enrig (deferasirox) dispersible tablets prepared according to this prescription meet the requirements.

The therapeutic effect of Deferasirox dispersible tablets is worthy of recognition. Enrig (deferasirox) dispersible tablets have 3 protruding ligands that bind to iron with high affinity in a ratio of 2:1. Although Deferasirox has a very low affinity for zinc and copper, serum concentrations of these trace metals decreased to varying degrees after administration.

The specific usage and dosage are: It is recommended for patients with iron accumulation, such as those who require long-term blood transfusion (blood transfusion volume reaches 100ml/kg) and whose serum ferritin exceeds 1000ug/liter; corresponding to the body weight, it is initially recommended to take a daily dose of 20mg/kg. For deferasirox, depending on the improvement of serum ferritin indicators, patients may need to adjust or increase the dose of deferasirox. Generally, the dose will be increased in units of 5 mg/kg or 10 mg/kg, but the total dose should not exceed 30 mg/kg per day.