地拉罗司治疗铁质积聚效果怎样？

Deferasirox is a tridentate iron chelator developed by Novartis and is currently the only oral iron chelator. Because Deferasirox continues to exist in plasma, it can continuously reduce plasma non-transferrin-bound iron and directly remove iron formed from toxicity in the body. So, how effective is Deferasirox in treating iron accumulation?

A multicenter, prospective clinical trial was conducted to evaluate the safety and efficacy of deferasirox (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 patients' feedback, Deferasirox (Enrig, Deferasirox) is very effective. It plays a good auxiliary role in patients with thalassemia or other rare anemias that require long-term blood transfusions that lead to iron accumulation (people who require long-term blood transfusions (blood transfusion volume up to 100 ml/kg) and serum ferritin exceeds 1000 μg/L).

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