地拉罗司的治疗效果怎么样呢？

People with chronic anemia such as thalassemia or sickle cell anemia often require regular red blood cell transfusions. Repeated infusions produce toxic and ultimately fatal iron accumulation in multiple tissues of the body as insoluble ferritin. This chronic iron overload occurs because the body cannot eliminate iron efficiently. Chronic iron overload is a serious symptom, and organ failure can occur due to the iron deposits produced. When the heart or liver is affected, the condition can be life-threatening. It is an oral iron chelator that 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, Deferasirox was approved by the European Commission for the treatment of chronic iron overload in patients aged 10 years and older 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 aged 10 years and older with non-transfusion-dependent thalassemia (NTDT).

The pharmacokinetics of deferasirox were studied and the efficacy of deferasirox was also verified. Five adult patients with thalassemia took 20 mg of deferasirox orally every day. 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 is through glucuronidation to generate the metabolites M3 (acyl glucuronide) and M6 (2-O-glucuronide).