地拉罗司对铁质积聚有多大的疗效？

Thalassemia, also known as thalassemia, is a hereditary disease that is very common in areas south of the Yangtze River in my country. If the disease is mild, it is generally anemia. But if the condition is more serious, it can directly threaten life. It is a disease that requires prompt treatment. At present, our best treatment for thalassemia is to use drugs produced by Novartis.

Deferasirox is the only oral iron chelator developed by Novartis. Deferasirox was approved by the FDA in November 2005 for use in patients 2 years of age 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.

So how effective is deferasirox on 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 DFO or deferasirox chelation therapy. 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.

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.