恩瑞格可以治疗什么病症呢？

(Deferasirox) is currently the only oral iron ion chelator developed by Novartis. What diseases can it treat?

In November 2005, the U.S. FDA approved Deferasirox for the treatment of 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 above with non-transfusion-dependent thalassemia (NTDT) syndrome who require chelation therapy due to contraindications or insufficiency of deferoxamine mesylate therapy. On January 23, 2013, the FDA approved a new indication for Deferasirox for the treatment of chronic iron overload in patients with non-transfusion-dependent thalassemia (NTDT) aged 10 years and above.

The common starting dose is 20 mg/(kg·d). If there is a large amount of blood transfusion and the iron increase is >0.5 mg/(kg·d) or the iron load before treatment is high, the recommended dose is 30 mg/(kg·d). If the iron increase is <0.3 mg/kg, 10-15 mg/kg/d is enough to control the disease. Administer on an empty stomach 30 minutes before meals and should be completely dissolved in water or apple or orange juice. Lower doses of Deferasirox [5-10 mg/(kg·d)] can only be used for young patients with mild iron overload and few blood transfusions, or elderly patients who have not received satisfactory results from chelation therapy in the past; patients using Deferasirox 20 and 30 mg/(kg·d) need to carefully monitor body iron stores, because body iron levels may continue to rise in 50% and 10% of patients. In addition, the frequency of blood transfusion can also affect the effect of Deferasirox in controlling liver concentrations. When the iron intake is greater than 0.5 mg/(kg·d), only 47% of patients who receive the same dose of Deferasirox achieve a reduction in liver concentrations.

Phase III clinical studies have observed that certain acute adverse reactions may occur with high-dose use of Deferasirox, such as gastrointestinal symptoms in 15% and rash in 11%, but patients who interrupt treatment due to these symptoms are rare. In 2008, the U.S. FDA received a report of a patient with encephalopathy who developed liver failure after taking Deferasirox for 5 days. The report revealed that the patient had a history of alcohol abuse and mild liver function abnormalities. In addition, the patient had no indication for medication, and his serum ferritin level was 10% of the medication indication recommended in the drug insert. The patient's liver function returned to normal after stopping the drug. Bauters et al. from Ghent University Hospital in Belgium reported a case of gastric ulcer in a child with thalassemia major after receiving Deferasirox treatment. According to the adverse events reported in recent years (Deferasirox), this case is the first one, so it requires sufficient attention.