Deferasirox是什么药？

Deferasirox (sold under the trade names Exjade, Desirox, Defrijet, Desifer, Rasiroxpine, and Jadenu) is an oral iron chelator. Its primary use is to reduce chronic iron overload in patients receiving chronic transfusions for conditions such as beta-thalassemia and other chronic anemias. This is the first oral drug approved for this purpose in the United States. It was approved by the U.S. Food and Drug Administration (FDA) in November 2005. According to the FDA (May 2007), renal failure and cytopenias have been reported in patients receiving deferasirox oral suspension tablets. It is approved in the EU by the European Medicines Agency (EMA) for patients aged 6 years and older with chronic iron overload from repeated blood transfusions.

The main indications of deferasirox are: 1. Patients with chronic iron overload and transfusion hemosiderinosis secondary to blood transfusion who are 2 years old or older will have symptoms of chronic iron overload when serum ferritin continues to be >1000 L, and iron chelation therapy should be started at this time. 2. Iron chelator therapy can also be given to patients receiving ASCT when iron overload occurs. 3. Can be used to treat reversible renal insufficiency caused by Fanconi syndrome.

The pharmacokinetics of deferasirox were studied. Five adult patients with thalassemia took 20 mg of deferasirox orally daily. After 7 days, the pharmacokinetics reached a stable state. Blood, plasma, feces, and urine samples were collected to analyze the concentrations of radioactive deferasirox, Fe-deferasirox complex, and deferasirox metabolites, and study the absorption, distribution, metabolism, and excretion processes of deferasirox. Studies have shown that deferasirox is well absorbed, with radioactive deferasirox and its Fe complex accounting for 87% and 10% of plasma concentrations, respectively, and most of them are excreted in the feces (84%), of which 60% is radioactive deferasirox. Unaltered deferasirox in the feces is partly due to incomplete intestinal absorption and partly due to hepatobiliary elimination of deferasirox and its glucuronide (including first-pass elimination), with renal excretion of only 8%, the main component being glucuronide M6. CYP450 enzymes catalyze deferasirox, and the products are M1 (5-OH deferasirox) (CYP1A) and M4 (5-OH deferasirox) (CYP2D6). Their contents are very small, accounting for only 6% and 2%. Direct and indirect results show that the main metabolic pathway of deferasirox is through glucuronidation to generate the metabolites M3 (acyl glucuronide) and M6 (2-O-glucuronide).