1.
Hematopoietic stem cell transplantation for children with thalassemia major in China.
Fang, JP, Xu, LH
Pediatric blood & cancer. 2010;(6):1062-5
Abstract
Thalassemia is the most common single-gene disorder worldwide that is considered a major public health issue. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for thalassemia major, the form of the disease reflecting homozygosity for a mutant allele. In China, many patients with thalassemia major cannot financially afford life-long regular supportive care with blood transfusions and iron chelation. Although HSCT is expensive, it is a one-time treatment that is possible in some patients. Disease-free survival rates have been 52-82% after HSCT in China. Graft rejection is the main cause of failure following HSCT. Humoral immune activity may play an important role in engraftment of donor cells. This article reviews the current status of HSCT for children with thalassemia major in China.
2.
Effects of silymarin on the proliferation and glutathione levels of peripheral blood mononuclear cells from beta-thalassemia major patients.
Alidoost, F, Gharagozloo, M, Bagherpour, B, Jafarian, A, Sajjadi, SE, Hourfar, H, Moayedi, B
International immunopharmacology. 2006;(8):1305-10
Abstract
Iron toxicity in beta-thalassemia major is the main cause of oxidative stress and cell mediated immune deficiencies. Despite indicative signs of severe oxidative deficiencies associated with beta-thalassemia major, such as decreased level of plasma antioxidants and depletion of erythrocyte glutathione, little is known about intracellular redox status of immune cells. Since glutathione is a primary intracellular antioxidant and plays an essential role in several functions in T cells, in this study intracellular glutathione (GSH) levels as well as proliferation of PHA-activated peripheral blood mononuclear cells (PBMC) were investigated in 28 beta-thalassemia major patients and 28 healthy age-matched individuals. Considering the potential benefits of flavonoids in the therapy of oxidative stress, the effects of silymarin on the GSH levels and proliferation of PBMC from normal and thalassemia individuals were further examined. Quantitative determination of intracellular GSH and proliferative response of PBMC to PHA were performed before and after 72 h incubation of PBMC with various concentrations of silymarin (0, 5, 10, or 20 mug/ml). Results demonstrated a significant reduction of GSH and proliferation in beta-thalassemia major cells; however treatment with silymarin led to restoration of both GSH levels and PBMC proliferation in thalassemia patients. Considerably low levels of GSH and depressed proliferative response of PBMC in beta-thalassemia major may be responsible for the cell mediated immune abnormalities in iron overload conditions. Moreover, the GSH restoration and improvement of PBMC growth by silymarin is a possible explanation for its recently reported antioxidant and immunostimulatory activities. These data suggest the benefit of using flavonoids to normalize immune dysfunction in beta-thalassemia major. The immunomodulatory effects of silymarin in beta-thalassemia major are currently under further investigation in a double blind clinical trial.