地拉罗司治疗铁质积聚的疗效如何呢？

The chemical name of (deferasirox) is 4-[3,5-bis(2-hydroxyphenyl)-1,2,4-triazol-1-yl]benzoic acid. It is an iron chelator product developed by the Swiss pharmaceutical company Novartis. It is the first oral iron-removing agent approved by the US FDA for routine use. It is approved for use in patients ≥2 years old with chronic iron overload caused by blood transfusion. In Europe, it is recommended as a 6 As a first-line drug for patients with thalassemia iron overload over the age of 18, clinical research is currently underway in China; Phase II and III clinical trials and pharmacokinetic studies have shown that it has good safety and tolerability, can significantly reduce iron load in the heart and liver, and is easily accepted by patients. At the same time, it also has pharmaceutical properties such as antifungal (such as Mucor that grows in an iron-rich environment), anti-cell proliferation, anti-malarial, anti-oxidative stress damage, anti-cytotoxic-induced apoptosis, etc.; it can be used for the treatment of secondary hemochromatosis, porphyria cutanea tarda and other diseases.

The benefits of iron removal therapy are not only reflected in the reduction of ferritin levels, but also can improve the patient's hematological response, stabilize and reverse the patient's liver fibrosis, improve the ejection fraction of the heart, and prevent the occurrence of important cardiac events such as heart failure and arrhythmia. my country's data also supports the above conclusion. The results of a single-arm, multi-center, prospective clinical study enrolling 64 AA patients with transfusion-related iron overload confirmed the effectiveness and safety of deferasirox in reducing patients' ferritin levels. A prospective, observational study in my country using liver, pancreas, and heart as monitoring indicators confirmed that deferasirox can improve organ function in patients with iron overload. The TELESTO study results released at ASH2018 also proved that deferasirox 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%.

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.