The Biological Characteristics of PNH Clones
YE Jinhui, WANG Xuan, WANG Shichong, PENG Xuemei, GE Meili, SHAO Yingqi, ZHENG Yizhou, CHENG Tao, HU Linping*
PNH (paroxysmal nocturnal hemoglobinuria) is an acquired clonal disease caused by the PIGA mutation in HSCs (hematopoietic stem cells), characterized mainly by hemolysis and bone marrow failure etc. In this study, flow cytometry and colony forming cell assays were performed to analyze the total number of blood cells and HSPCs in bone marrow and peripheral blood from cPNH patients compared with health donors. Moreover, the characteristics of PNH clones were investigated. The results revealed that the number of T cells, B cells and NK cells in peripheral blood from cPNH decreased significantly, while no significant difference was observed in the number of myeloid cells compared with health donors. Among many kinds of blood cells in bone marrow, the proportion of monocytes in cPNH was significantly higher than that in the control group, while there was no significant difference in the proportion of neutrophils, nucleated erythrocytes and lymphoid cells between cPNH and control group. All the HSPCs from cPNH bone marrow were abnormal PNH clones detected as CD59–. Moreover, there was an extensive exhaustion of HSPCs in cPNH, and their lymphoid differentiation potential was obviously inhibited, which was consistent with the change of B cells and NK cells in peripheral blood and bone marrow. These data indicated that lymphoid cells were the most severely impaired cells in cPNH. CFU assays manifested that there was no difference in myeloid colony forming ability between control and cPNH. As for the erythroid colony, cPNH HSPCs formed more small erythroid colonies for compensation. In conclusion, this study showed the biological characteristics of PNH clones, which provided theoretical basis for the diagnosis and treatment of PNH.
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