Department of Pediatric Hematology and Oncology, Mofid Children Hospital, Shahid Beheshti University of Medical Science, Tehran.
Daru. 2011;19(3):240-8.
to determine the efficacy, adverse effects and safety of a new Iranian generic product of deferasirox (Osveral®) in Iranian transfusion dependent major thalassemic (TD-MT) patients.
In 9 main thalassemia treatment centers, all of TD-MT patients (aged ≥2 yrs) with serum ferritin (SF) levels≥1000 ng/ml, or >100 ml/kg of RBC transfusion,who could not tolerate parental iron chelating were recruited regardless of their previous iron chelation therapy. Periodical clinical and laboratory evaluations were conducted for adverse effects (AEs). Primary efficacy end point was Mean of Relative Change of Serum Ferritin (MRC-SF) from the baseline level during one year. Analysis of variance (ANOVA), t test, chi-square or Fisher exact test were used for statistic analysis appropriately (P values <0.05 were considered as statistical significant).
In 407 cases the male/female ratio was 0.98. Mean age was 11.5±7.4 (2-58) years. The mean of initiating dose of Osveral® and mean usage dose during the study was 23.5±4.9 mg/kg and 24.9±4.9 mg/kg respectively. MRC-SF was -11.44% ±38.92 and it showed significant decline in SF (P value<0.001) one hundred and forty eight patients out of 407 patients experienced at least one. AE, the most common of them were transient increase in serum creatinin (97;24.1%) and>5 time increase in transaminases (24;5.89%).The causes of discontinuation of treatment were non-satisfactory treatment ( 24; 5.8%), poor or non-compliance of patients (21;5.1%), and adverse effects (13; 3.1%)
A detailed comparison with similar studies on deferasirox (Exjade®) shows a promising efficacy and safety for its Iranian generic product (Osveral ®).
确定一种新的伊朗仿制药去铁酮（Osveral®）在伊朗输血依赖型重型地中海贫血（TD-MT）患者中的疗效、不良反应和安全性。
在 9 个主要的地中海贫血治疗中心，所有血清铁蛋白（SF）水平≥1000ng/ml或>100ml/kg RBC 输血的 TD-MT 患者（年龄≥2 岁），且不能耐受亲体铁螯合剂，无论其先前的铁螯合治疗如何，均被招募入组。定期进行临床和实验室评估以观察不良反应（AE）。主要疗效终点为治疗 1 年时血清铁蛋白相对变化均值（MRC-SF）。适当采用方差分析（ANOVA）、t 检验、卡方检验或 Fisher 确切检验进行统计分析（P 值<0.05 认为具有统计学意义）。
407 例患者中，男女比例为 0.98。平均年龄为 11.5±7.4（2-58）岁。Osveral®起始剂量和研究期间的平均使用剂量分别为 23.5±4.9mg/kg和 24.9±4.9mg/kg。MRC-SF 为-11.44%±38.92，SF 显著下降（P 值<0.001）。407 例患者中有 148 例出现至少 1 种不良反应（AE），最常见的是血清肌酸酐一过性升高（97 例；24.1%）和转氨酶升高>5 倍（24 例；5.89%）。治疗中断的原因包括治疗不满意（24 例；5.8%）、患者情况不佳或不依从（21 例；5.1%）和不良反应（13 例；3.1%）。
与去铁酮（Exjade®）的类似研究进行详细比较后表明，其伊朗仿制药（Osveral®）具有良好的疗效和安全性。
