부산대학교 도서관

Background and Objective Increased expression of fetal hemoglobin ( Hb F) may ameliorate the clinical course of hemoglobinopathies like sickle cell disease ( SCD) and β-thalassemia. Hydroxyurea ( HU) can stimulate Hb F production in these diseases but the response is highly variable indicating the utility of developing an in vitro test to predict the patient's response to HU. We assessed whether the Hb F response of patients with SCD and thalassemia intermedia ( TI) to HU correlates with HBG (both γ-globin genes) expression in their cultured erythroid progenitors following exposure to HU. Patients and Methods We exposed primary erythroid cultures from peripheral blood mononuclear cells from 30 patients with SCD and 15 with TI to HU and measured HBG m RNA by real-time quantitative PCR. The same patients were then treated with HU and their Hb F response after treatment with a stable dose of HU was compared with the m RNA results in cultured cells. Results and Conclusion The fold increase in HBG m RNA in erythroid progenitors was similar to the fold increase in Hb F in vivo. Quantification of HBG m RNA in erythroid progenitor cell cultures from patients with SCD and TI is predictive of their clinical response to HU. [ABSTRACT FROM AUTHOR]