Study On The Characteristics Of The Hematopoietic Stem/progenitor Cells Of Paroxysmal Nocturnal Hemoglobinuria Patients

OBJECTTIVES:1 .To observe the efficacy and side effects of DA/HA chemotherapy on refractoryand relapsed paroxysmal nocturnal hemoglobinuria (PNH).2.To compare the growth and the response of PNH and normal controls' bonemarrow mononuclear cells (BMMNCs) to the granulocyte colony-stimulatingfactor (G-CSF) in vitro.3.To study the expression of G-CSF receptor (G-CSFR)(CD114) and stem cellfactor (SCF) receptor (SCFR)(CD117) on BMMNCs of PNH patients and normalcontrols.4.To analyze normal and PNH patients' bone marrow (BM) neutrophils forCD16b, apoptotic receptor Fas (CD95) and apoptosis related proteins Bcl-2 andBax.METHOD:1.Clinical trial:8 patients with refractory and relapsed PNH were treated with DA/HA chemotherapy. The regimens were as following: 3 patients were treated with DA (DNR40 mg/d, i. v. drip, the first and the second day; 20mg/d, i. v. drip, the third day; Ara-C 100mg/d, i. v. drip, for 5 days), 5 patients were treated with HA (HHT 2mg/d~3mg/d, i.v.drip, for 5 days; Ara-C 100mg/d, i.v.drip, for 5 days). The therapeutic effects were observed.2.In vitro culture:BMMNCs from17 PNH patients and 12 normal controls were inoculated into semisolid culture, divided into two groups: one growed with G-CSF (50ng/ml), the other without. Then the cluster/colony forming unit-granulocyte/monocyte (CFU/cFU-GM) were observed and the effect of G-CSF were compared.3.Mechanism investigation:(1)BMMNCs of 20 PNH patients and 12 normal controls were triple stained for...