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Paroxysmal nocturnal hemoglobinuria/aplastic anemia (PNH/AA) syndrome presents a markedly increased population of cells deficient in glycophosphatidylinositol (GPI - cells) and signs of bone marrow failure, which requires treatment with hematopoiesis-stimulating factors, such as granulocyte colony-stimulating factor (G-CSF) and stem cell factor (SCF). However, little is known about the effects of these stimulating factors on GPI - cells. In order to explore the effects of stimulating factors in PNH/AA, G-CSF receptor (CD114) and SCF receptor (CD117) expression levels on GPI + and GPI - hematopoietic stem cells (HSCs) were measured by flow cytometry (FCM). The mean fluorescence intensity (MFI) values of signal transducer and activator of transcription 5 (STAT5) and phosphorylated (P)-STAT5 were measured in GPI + and GPI - HSCs by FCM following stimulation with G-CSF or SCF in vitro . The expression levels of CD114 and CD117 on GPI - HSCs were significantly lower (P&lt;0.01) than those on GPI + HSCs in PNH/AA patients and normal controls. The MFI values of STAT5 in the GPI - and GPI + HSCs of PNH/AA patients and normal controls were not significantly different. However, the MFI values of P-STAT5 in the GPI - HSCs of PNH/AA patients were significantly lower than those in the GPI + HSCs of PNH/AA patients and normal controls prior to and following stimulation with G-CSF or SCF (P&lt;0.01). The GPI - HSCs of PNH/AA patients responded poorly to stimulation by hematopoiesis-stimulating factors, which indicates that these factors can be used safely in patients with PNH/AA.

experimental and therapeutic medicine

Expression and function of hematopoiesis-stimulating factor receptors on the GPI− and GPI+ hematopoietic stem cells of patients with paroxysmal nocturnal hemoglobinuria/aplastic anemia syndrome