恩瑞格治疗铁质积聚效果如何呢？

(Deferasirox) is an oral iron chelator that was approved by the FDA in 2005 for the treatment of chronic iron overload caused by blood transfusions. The original research was by Novartis Pharmaceuticals. In January 2013, the FDA approved expanded indications to treat chronic iron overdose caused by Non-Transfusion Dependent Thalassemia Syndromes (NTDT), making Exjade the first drug to treat this indication. NTDT is a mild form of thalassemia that does not require frequent blood transfusions. However, even if patients do not receive blood transfusions, they will accumulate excess iron and damage various tissues and organs. Currently, deferasirox has been approved in more than 100 countries around the world for the treatment of transfusion-induced iron overload in children over 2 years of age. In China, only Deferasirox, the original product of Novartis Pharmaceuticals, was launched in June 2010, and only Jiangsu Aosaikang Pharmaceutical is applying for a domestically produced generic version.

So how effective is Enrig in treating iron accumulation?

According to a retrospective study in Italy, 42.7% of patients achieved a hematological response and reduced the need for blood transfusions after receiving chelation therapy with DFO or Deferasirox. There may be a dynamic, bidirectional regulatory mechanism between erythropoiesis and iron overload. On the one hand, ineffective erythropoiesis can lead to increased intestinal iron absorption through reduced hepcidin secretion by the liver. On the other hand, chelation treatment of iron overload can improve erythropoiesis, increase hemoglobin levels, and reduce clinical blood transfusions.

Adverse reactions of Deferasirox: (1) Diarrhea, vomiting, headache, abdominal pain, fever, rash, increase in serum creatinine (Creatinine), etc. (2) Certain acute adverse reactions may occur when using large doses of deferasirox, such as gastrointestinal symptoms (15%) and rash (11%). However, it is rare for patients to discontinue treatment due to these symptoms. (3) For patients with renal insufficiency, Deferasirox can cause an increase in creatinine levels, and 38% of patients have an increase in creatinine levels of more than 33%. However, the increase in creatinine levels caused by Deferasirox generally does not exceed the upper limit of normal and has not been reported to cause progressive kidney disease. After dose reduction of Deferasirox, creatinine levels returned to normal or remained stable in 13% of patients.