Deferasirox疗效怎么样呢？

It is clinically used for patients with iron accumulation due to long-term blood transfusion (such as those suffering from thalassemia or other rare anemias). It is a new type of ferric iron chelator. So, what is the efficacy of Deferasirox?

Therapeutic effects of Deferasirox:

A clinical trial 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 and 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.

It is very effective and plays a good auxiliary role in patients with thalassemia or other rare anemias that require long-term blood transfusions resulting in iron accumulation (people who require long-term blood transfusions (blood transfusion volume up to 100 ml/kg) and whose serum ferritin exceeds 1000 μg/L).