瑞士诺华的地拉罗司去铁效果如何？

How effective is Novartis's iron removal treatment?

Deferasirox is used to treat iron overload caused by blood transfusions in patients with certain hematologic disorders. In 2005, deferasirox was approved by the FDA for the treatment of iron overload caused by blood transfusions during treatment in patients with chronic anemia 2 years of age and older.

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. According to the International Working Group's 2006 criteria, 51 patients (28%) had improved hemograms, of which only 7 patients received growth factors or MDS. During the 3-year study, 138 (79.8%) patients discontinued treatment, 43 (24.8%) of whom discontinued treatment due to adverse events or disease progression, and 23 (13.2%) due to abnormal laboratory test values.

The results of this trial show that it can reduce serum ferritin and LPI in transfusion-dependent MDS patients, and some patients' hemogram and liver function can be improved to a certain extent.

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