恩瑞格的效果好吗？

Deferasirox dispersible tablets () are mainly suitable for patients with iron accumulation due to long-term blood transfusion (such as those suffering from thalassemia or other rare anemias). It is suitable for children over two years old and adults. Enriga should not be used with other iron chelation treatments as the safety of such combined use has not been established. Enrig is contraindicated in patients with creatinine clearance <40mL/min or serum creatinine >2 times the upper limit of normal for the corresponding age; patients with poor general condition, high-risk myelodysplastic syndrome (MDS) or advanced malignant tumors; patients with platelet count <50x109/L.

Is Enrig 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).