Mini-pool intravenous immunoglobulin G was well tolerated, presented no safety issues, and was effective in the treatment of immune thrombocytopenia, with efficacy comparable to that of the standard intravenous immunoglobulin G group, and it was significantly more effective than no treatment.
Background: Metabolic challenges in beta thalassemia major (BTM) carry an additional burden to both chronic anemia and iron overload. Although optimal nutritional support for children with BTM is mandatory, health insurance cover only blood transfusion and chelation therapy. Nutrition support remains subtle for both the family and governmental interest especially in developing countries. Aim: To assess growth, total body composition, lipid and glucose hemostasis in Egyptian children with BTM in relation to iron overload and state of chronic hypoxemia. Subjects and methods: 200 Egyptian children with BTM were recruited, they were 99 females (49.5%) and 101 male (50.5%) and their mean age was 12.15 ± 4.23 years. 50 age- and sex-matched healthy children with same socio-economic background were also enrolled. All of them subjected to history taking; thorough clinical examination, anthropometric measurements (weight, height, mid arm circumference, skin fold thickness and body mass index), body composition analysis (using bioelectric impedance analysis device) and dietetic assessment (using 24-hours dietetic recall). Serum lipid profile (high-density lipoprotein (HDL), low-density lipoprotein (LDL), cholesterol and triglyceride), C-peptide and serum insulin were assessed. Results: Most children with BTM had families with high birth order and nearly half of them had positive family history, in addition consanguinity was reported to be 1.5 times in BTM compared to what was reported in controls. Almost half of the patients were from Cairo, the other half were from the economically less privileged Upper Egypt. Most of patients were diagnosed presented in 1st two years of life at mean age of 1.52 ± 1.66 and 1st transfusion was initiated within first four months after initial diagnosis. Most of patient transfused on low mean pre-transfusion hemoglobin of 8.4± 2.6 g/dl. Most studied BTM had iron overload, median (IQR) of serum ferritin was 1800 (1149 - 3100) ng/ml in the last 2 years, with median (IQR) duration on regular iron chelation was 4 (2-6) years and 12.5% of patients were non-compliant. Children with BTM had a significantly lower nutritional intake (kilocalories, protein, carbohydrate, calcium, and phosphorus) than controls with negative impact on anthropometric parameters and body composition parameters both in pubertal and pre-pubertal stage. There was significant increase in body fat value and percent in female BTM compared to male BTM. While there was significant increase in muscle mass % and body water % in males BTM. Children with BTM had significantly higher triglycerides levels however lower cholesterol, LDL, HDL, insulin and C-peptide compared to control. Factors including age, gender, caloric intake, mean pre-transfusion hemoglobin, transfusion burden, splenectomy, viral hepatitis comorbidity, mean serum ferritin, serum insulin, and HDL have impact on body composition in children with BTM. Conclusion: Poor caloric intake with low pre-transfusion hemoglobin and higher serum ferritin were the main risk factors for poor anthropometric parameters in all ages. Regular assessment of nutrition is crucial for the health of children with thalassemia. Keywords: Thalassemia, Body composition; Nutritional deficiency; Diabetes; Lipid Disclosures No relevant conflicts of interest to declare.
Introduction: Oxidative stress plays a central role in the pathogenesis of morbidities in β-thalassemia. Peroxiredoxin-2 (PRDX2) is the third most abundant cytoplasmic protein in red cells and is able to reduce and detoxify a vast range of organic peroxides, H2O2, and peroxynitrite. PRDX2 has been demonstrated to be induced by oxidative stress and that cells overexpressing PRDX2 are more resistant to the oxidative stress. Vitamin E is a fat-soluble vitamin and an anti-oxidant which is often depleted in thalassemia patients as a result of iron overload. Aim: This randomized prospective trial aimed to investigate the efficacy and safety of vitamin E as an adjuvant therapy to the three used iron chelators in moderately iron overloaded young vitamin E-deficient β-thalassemia major (β-TM) patients in relation to tissue iron overload and examine its potential corrective value to markers of oxidative stress and to the level of PRDX2 as a novel protective marker of oxidative stress. Methods: Inclusion criteria were β-TM patients aged 6-18 years, vitamin E deficient, serum ferritin (SF) >1000-2500 μg/L and cardiac T2* > 10 ms and ejection fraction > 56 %. A total of 245 β-TM patients were screened for eligibility, 180 were enrolled while 35 did not meet inclusion criteria and 30 were excluded. The 180 β-TM vitamin E-deficient patients were equally enrolled into 3 groups (each; n=60) and received desferrioxamine (DFO), deferiprone (DFP) and deferasirox (DFX). Patients in each group were further randomized either to receive vitamin E supplementation (400 IU daily) or not (n=30). All patients received vitamin E (group A) or no vitamin E (group B) were followed-up for one year with assessment of transfusion index, hemoglobin, serum ferritin (SF), liver iron content (LIC) and cardiac magnetic resonance imaging (MRI) T2*. Malondialdehyde (MDA), as an index for lipid peroxidation, and antioxidants including reduced glutathione (GSH), superoxide dismutase (SOD), glutathione peroxidase (GPX), catalase and PRXD2 were assessed. Results: Baseline vitamin E was negatively correlated to SF, LIC and MDA while it was positively correlated to GSH, GPX, catalase and PRXD2. No significant difference was found as regards the studied variables at baseline and study end in patients who did not receive vitamin E (group B). After vitamin E therapy, transfusion index, SF, and LIC were significantly decreased in group A patients while hemoglobin and cardiac MRI T2* were elevated compared with baseline levels or those in group B without vitamin E. MDA levels were decreased while all the studied antioxidants were increased post-vitamin E supplementation compared with baseline levels or those in group B without vitamin E. The same improvement was found among DFP-treated patients post-vitamin E compared with baseline data. DFP-treated patients had the highest hemoglobin and antioxidant levels with the lowest SF and LIC compared with DFO or DFX subgroups. Conclusions: Vitamin E as an adjuvant therapy possibly potentiates the efficacy of DFP more than DFO and DFX in reducing iron burden and reduces oxidative stress in the moderately iron overloaded vitamin E-deficient β-TM patients, with no adverse events. Disclosures No relevant conflicts of interest to declare.
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