地拉罗司治疗铁质积聚效果如何呢？

It is a drug approved to reduce iron burden. So, how effective is deferasirox in treating iron accumulation?

Therapeutic effects of deferasirox in treating iron accumulation:

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 most common drug-related adverse events were gastrointestinal disturbances and increased serum creatinine. Twenty-eight patients died, but none were related to deferasirox treatment.

The results of this trial show that deferasirox 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.

In 2005, it was approved by the FDA for the treatment of iron overload caused by blood transfusions during treatment in patients with chronic anemia 2 years and older.