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

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]

[Predictive value of nucleated red blood cell counts in cord and peripheral blood of asphyxiated term neonates in the first week of life]

Increased numbers of nucleated red blood Cells [NRBC] circulating in the blood of neonates can be associated with relative hypoxia and adverse outcomes. Thus, the aim of this study was to assess the NRBC count during the first week of life in neonates diagnosed with asphyxia as compared to healthy neonates and to determine the short-term morbidity and mortality for the affected babies. The cross-sectional study compared 15 healthy neonates with 15 neonates diagnosed with asphyxia confirmed by pH of cord blood or Apgar scores. The nucleated red blood cell [NRBC] counts were calculated right after birth, and on days 3 and 7, and the hematological parameters of umbilical cord blood were also evaluated. The infants were followed for mortality and associated morbidity. Statistical analysis was conducted using the Mann-Whitney U test, analysis of variance, chi-square tests, and Pearson's correlation coefficient. A p- value <0.05 was considered as statistically significant. The initial NRBC counts were significantly higher in the asphyxiated group than in the control group and the difference remained significant through the end of first week. All of the umbilical cord blood parameters were significantly lower in the study group and were negatively correlated with the NRBC count. At birth, higher NRBC count correlated with higher mortality. Results show that NRBC count is a useful predictive factor for neonatal asphyxia through the end of the first week of life, although a larger study population and a longer follow up period seems to be necessary

cross-sectional controlled study of gonadal function and pubertal development in thalassemia major

Early and regular blood transfusion therapy in patients with homozygous beta-thalassemia decreases the complications of severe anemia and prolongs survival. In the long term, however, the beneficial effects of transfusions are limited by the organ damage resulting from iron overload. Endocrine complications in patients with thalassemia major in developing countries may be frequent due to suboptimal iron chelation. The goal of this study was to investigate the gonadal function and secondary sex characteristics in thalassemic patients. We studied 71 randomly selected adolescent thalassemic patients and 30 age- and sex-matched controls. Sexual maturity rating [SMR], height and weight, and gonadotropin, sex steroid, and ferritin levels were evaluated. Cases had significantly lower mean height and weight. Also, serum levels of gonadotropins and sex hormones were significantly lower in cases with thalassemia than in controls. Gonadotropin and sex steroid levels were lower in cases with thalassemia who had not used deferoxamine regularly compared to those with a regular chelation therapy regimen. All of the control subjects had sexual maturity ratings of II or above, while 36.6% of thalassemic cases were in Tanner stage I. About 53.3% of controls had surpassed all levels of sexual maturity, while only 2.8% of cases were in the stage SMR V. Distribution of SMR ratings was significantly different in cases and controls. These findings clearly show that a high percentage of thalassemic patients in this part of the country suffer from various endocrine abnormalities, especially impairment in height, growth and sexual maturity. It is wise to consider more sophisticated treatment modalities in these patients, including the administration of sex hormones for the compensation of hormonal abnormalities resulting from hemosiderosis