地拉罗司(恩瑞格)对铁质积聚的疗效

(Deferasirox) is an oral iron chelator approved by the FDA for the treatment of chronic iron overload caused by blood transfusion. Deferasirox is from Novartis. Later, the FDA approved the expanded indication of deferasirox to treat chronic iron overdose caused by Non-Transfusion Dependent Thalassemia Syndromes (NTDT), making deferasirox the first drug to treat this indication.

A multicenter, prospective clinical trial was conducted to evaluate the safety and efficacy of deferasirox (Deferasirox) in patients with low-risk or intermediate-risk-1 myelodysplastic syndrome (MDS). The study selected patients with serum ferritin ≥1000 μg/L, transfusion dependence and who had received red blood cell transfusions ≥20U. 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). 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.