地拉罗司疗效好吗？

It is currently the only oral iron chelator developed by Novartis. It was approved by the FDA in November 2005 for use in patients 2 years and older with chronic iron overload caused by blood transfusions. In December 2012, Deferasirox was approved by the European Commission for the treatment of chronic iron overload in patients aged 10 years and older with non-transfusion-dependent thalassemia (NTDT) syndrome who require chelation therapy due to contraindications or insufficiency of deferoxamine mesylate therapy. Enreg is a drug commonly used to relieve iron accumulation.

So is deferasirox effective?

This 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.

According to the patients' response, the efficacy is very good. It 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).