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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 First-line drug for patients with thalassemia and iron overload over 10 years of age.

A multicenter, prospective clinical trial was conducted to evaluate the safety and efficacy of deferasirox in patients with low- or intermediate-risk-1 myelodysplastic syndrome (MDS).

The study selected patients with serum ferritin ≥1000 μg/L, transfusion dependence and red blood cell transfusions ≥20U to be included. The starting dose of deferasirox is 20 mg/kg/d, which can be adjusted up to 40 mg/kg/d.

A total of 176 patients were enrolled in the study, of whom 173 received treatment. 53% of patients completed 12 months of treatment (n = 91) with a 23% decrease in mean serum ferritin, 36.7% completed 2 years of treatment (N = 49), and 36.5% completed 3 years of treatment (N = 33).

The study found that the reduction of serum ferritin was significantly positively correlated with the improvement of ALT (P <0.001). Baseline labile plasma iron (LPI) was elevated in 68 patients (39.3%). After 13 weeks, all patients with abnormal baseline LPI levels returned to normal.

It is recommended for 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. Based on the body weight, it is recommended to take a daily dose of 20 mg/kg in the initial stage.