Efficacy and safety of Iranian made deferasirox [Osveral]in Iranian major thalassemic patients with transfusional iron overload: a one year prospective multicentric open-label non-comparative study

ABSTRACT

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]