Department of Hematology/Oncology, Santa Casa Medical School, São Paulo, Brazil.
Acta Haematol. 2012;128(2):113-8. doi: 10.1159/000338560. Epub 2012 Jun 30.
The efficacy and safety of a 2-year treatment with deferasirox was evaluated in 31 patients with sickle cell anemia and transfusional iron overload. At 24 months, there were significant decreases from baseline in mean serum ferritin (from 2,344.6 to 1,986.3 µg/l; p = 0.040) and in mean liver iron concentration (from 13.0 ± 5.4 to 9.3 ± 5.7 mg Fe/g dry weight; p < 0.001). Myocardial T2* values were normal (>20 ms) in all patients at baseline and did not change significantly over the course of the study. However, there was a significant improvement from baseline in left ventricular ejection fraction at 24 months (62.2-64.6%; p = 0.02). Deferasirox was generally well tolerated with no progressive increases in serum creatinine or renal failure observed. These data confirm that deferasirox is effective in reducing body iron burden in patients with sickle cell anemia and transfusional iron overload.
31 例镰状细胞贫血伴输血铁过载患者接受了为期 2 年的地拉罗司治疗，评估其疗效和安全性。24 个月时，血清铁蛋白均值（从 2344.6 降至 1986.3μg/l；p=0.040）和肝脏铁浓度均值（从 13.0±5.4 降至 9.3±5.7mg Fe/g 干重；p<0.001）均较基线显著下降。所有患者的心肌 T2*值在基线时均正常（>20ms），研究过程中无明显变化。然而，左心室射血分数在 24 个月时较基线显著改善（62.2%-64.6%；p=0.02）。地拉罗司总体耐受性良好，未观察到血清肌酐持续升高或肾功能衰竭。这些数据证实，地拉罗司可有效降低镰状细胞贫血伴输血铁过载患者的体内铁负荷。
