恩瑞格的疗效怎样呢？

It is an oral iron chelator. Enrig was approved by the FDA in 2005 for the treatment of chronic iron overload caused by blood transfusion. Enrig is from Novartis. In January 2013, the FDA approved the expanded indication of Enrig to treat chronic iron overdose caused by Non-Transfusion Dependent Thalassemia Syndromes (NTDT), making Enrig the first drug to treat this indication.

The recommended starting daily dose of Enrig is 20 mg/kg. For patients who receive more than 14 mL/kg of packed red blood cells per month (i.e., more than 4 units/month for adults) and need to reduce excess iron exposure, a starting dose of 30 mg/kg/day may be considered; for patients who receive less than 7 units per month Patients who require mL/kg packed red blood cell (i.e., less than 2 units/month for adults) transfusions and who need to maintain iron balance in the body may consider an initial dose of 10 mg/kg/day; patients who have already responded well to deferoxamine treatment may consider an initial Enrig dose equivalent to half the deferoxamine dose. (For example, a patient receiving deferoxamine 40 mg/kg/day, 5 days per week, or equivalent, could start at 20 mg/kg if switched to Enriga.

The results of a single-arm, multi-center, prospective clinical study involving 64 AA patients with transfusion-related iron overload confirmed the effectiveness and safety of Enrig in reducing patients' ferritin levels. A prospective, observational study in my country using liver, pancreas, and heart as monitoring indicators confirmed that Enrig can improve organ function in patients with iron overload. The results of the TELESTO study released at the ASH 2018 meeting also proved that Enrige continuously reduces serum ferritin levels, while reducing the risk of heart function damage, liver function damage, transformation to leukemia and other events by 36.4%. EPIC is currently the largest prospective, open, single-group, global multi-center clinical study designed for patients with iron overload. A total of 341 MDS patients were enrolled, and it could reduce serum ferritin by an average of 372 μg/L in 3 months. 

In summary, iron overload increases the risk of infection, endocrine system damage, vital organ dysfunction, AML transformation in MDS patients, and shortens survival. Iron chelators are currently the mainstream treatment for iron removal. Among them, it is convenient to administer, significantly reduces ferritin levels, and has a definite effect in protecting important target organ systems such as the liver, heart, and endocrine system. It is currently the preferred option for iron removal treatment.