地拉罗司治疗铁质积聚疗效如何？

Also known as Enrig, it is mainly used to treat chronic iron overload caused by frequent blood transfusions (monthly administration of concentrated red blood cells ≥7mL/kg) in patients with β-thalassemia older than 6 years old. For children under 6 years old and iron overload caused by other transfusion-dependent diseases, how effective is deferasirox in treating iron accumulation?

Deferasirox is more convenient and has higher patient compliance. 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.

More importantly, the benefits of iron removal therapy are not only reflected in the reduction of ferritin levels, but can also improve the patient's hematological response, stabilize and reverse the patient's liver fibrosis, improve the heart's ejection fraction, and prevent the occurrence of important cardiac events such as heart failure and arrhythmia. Our 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%.

It is recommended that patients with iron accumulation, such as those who require long-term blood transfusion (blood transfusion volume up to 100 ml/kg) and whose serum ferritin exceeds 1000 μg/L, are initially recommended to take deferasirox at a dose of 20 mg/kg daily depending on their body weight. Depending on the improvement of serum ferritin indicators, patients may need to adjust or increase the dose of deferasirox. Generally, the dose will be increased in units of 5 mg/kg or 10 mg/kg, but the total dose should not exceed 30 mg/kg per day.

Dispersible tablets may cause gastrointestinal disorders, rash, mild elevation of serum creatinine, transaminases and other problems. Therefore, it is recommended that patients take medicines under the guidance of a doctor and avoid overuse. Deferasirox dispersible tablets have very low affinity for zinc and copper, but the concentrations of these trace metals in serum after administration still vary.