Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome
Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome
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DOI：
10.1182/blood-2002-03-0799
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发表时间：
2002-12-01
期刊：
影响因子：
20.3
通讯作者：
Nakao, S
中科院分区：
文献类型：
--
作者：
Wang, HB;Chuhjo, T;Nakao, S
在再生障碍性贫血（AA）患者中经常发现少量缺乏糖基磷脂酰肌醇（GPI）锚定膜蛋白的血细胞，尽管这种阵发性夜间血红蛋白尿（PNH）型细胞的临床意义尚不清楚。为了澄清这一问题，我们使用灵敏流式细胞术研究了164例骨髓增生异常综合征（MDS）患者CD55(-)CD59（-）粒细胞和红细胞的存在。在MDS的不同亚组中，119例难治性贫血（RA）患者中有21例检测到pnh型细胞显著增加（即至少0.003%）；PNH型细胞增加的RA患者（PNH+患者）的频率（17.6%）远低于我们之前报道的AA患者（52.0%）。与无PNH型细胞增加的RA患者（PNH-患者）相比，PNH+ RA患者具有明显的临床特征，如红细胞形态异常不明显，血小板减少更严重，核型异常发生率（4.8%对32.8%）和进展为急性白血病的发生率（0%对6.2%）较低，环孢素治疗反应的概率较高（77.8%对0%），HLA-DR15发生率较高（90.5%对18.5%）。这些数据表明，少量pnh型细胞的存在提示良性骨髓衰竭，可能是由免疫机制引起的。为了选择合适的治疗方法，治疗前应使用流式细胞术检测所有骨髓衰竭患者外周血中pnh型细胞的存在。(C) 2002年由美国血液病学会出版。
A minor population of blood cells deficient of glycosylphosphatidylinositol (GPI)-anchored membrane proteins is often detected in patients with aplastic anemia (AA), though the clinical significance of such paroxysmal nocturnal hemoglobinuria (PNH)-type cells remains unclear. To clarify this issue, we studied 164 patients with myelodysplastic syndrome (MDS) for the presence of CD55(-)CD59(-) granulocytes and red blood cells using sensitive flow cytometry. Among the different subgroups of MDS, a significant increase (ie, at least 0.003%) of PNH-type cells was detected in 21 of 119 patients with refractory anemia (RA); this frequency (17.6%) of RA patients with increased PNH-type cells (PNH+ patients) was much lower than what we previously reported (52.0%) for AA patients. PNH+ RA patients had distinct clinical features compared with RA patients without increased PNH-type cells (PNH- patients), such as less pronounced morphologic abnormality of blood cells, more severe thrombocytopenia, lower rates of karyotypic abnormality (4.8% vs 32.8%) and of progression to acute leukemia (0% vs 6.2%), higher probability of response to cyclosporine therapy (77.8% vs 0%), and higher incidence of HLA-DR15 (90.5% vs 18.5%). These data indicate that the presence of a minor population of PNH-type cells suggests a benign type of bone marrow failure, probably caused by an immunologic mechanism. To choose an appropriate therapy, peripheral blood should be tested using sensitive flow cytometry for the presence of PNH-type cells in all patients with bone marrow failure before treatment. (C) 2002 by The American Society of Hematology.