阵发性睡眠性血红蛋白尿症克隆在接受马抗胸腺细胞球蛋白加环孢素治疗的重型再生障碍性贫血患者中出现。
Paroxysmal nocturnal hemoglobinuria clones in severe aplastic anemia patients treated with horse anti-thymocyte globulin plus cyclosporine.
机构信息
Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, 20892-1202, USA.
出版信息
Haematologica. 2010 Jul;95(7):1075-80. doi: 10.3324/haematol.2009.017889.
BACKGROUND
Clones of glycosylphosphatidylinositol-anchor protein-deficient cells are characteristic in paroxysmal nocturnal hemoglobinuria and are present in about 40-50% of patients with severe aplastic anemia. Flow cytometry has allowed for sensitive and precise measurement of glycosylphosphatidylinositol-anchor protein-deficient red blood cells and neutrophils in severe aplastic anemia.
DESIGN AND METHODS
We conducted a retrospective analysis of paroxysmal nocturnal hemoglobinuria clones measured by flow cytometry in 207 consecutive severe aplastic anemia patients who received immunosuppressive therapy with a horse anti-thymocyte globulin plus cyclosporine regimen from 2000 to 2008.
RESULTS
The presence of a glycosylphosphatidylinositol-anchor protein-deficient clone was detected in 83 (40%) patients pre-treatment, and the median clone size was 9.7% (interquartile range 3.5-29). In patients without a detectable clone pre-treatment, the appearance of a clone after immunosuppressive therapy was infrequent, and in most with a clone pre-treatment, clone size often decreased after immunosuppressive therapy. However, in 30 patients, an increase in clone size was observed after immunosuppressive therapy. The majority of patients with a paroxysmal nocturnal hemoglobinuria clone detected after immunosuppressive therapy did not have an elevated lactate dehydrogenase, nor did they experience hemolysis or thrombosis, and they did not require specific interventions with anticoagulation and/or eculizumab. Of the 7 patients who did require therapy for clinical paroxysmal nocturnal hemoglobinuria symptoms and signs, all had an elevated lactate dehydrogenase and a clone size greater than 50%. In all, 18 (8.6%) patients had a clone greater than 50% at any given time of sampling.
CONCLUSIONS
The presence of a paroxysmal nocturnal hemoglobinuria clone in severe aplastic anemia is associated with low morbidity and mortality, and specific measures to address clinical paroxysmal nocturnal hemoglobinuria are seldom required.
背景
糖基磷脂酰肌醇锚蛋白缺陷细胞的克隆在阵发性睡眠性血红蛋白尿症中具有特征性，并且存在于约 40-50%的严重再生障碍性贫血患者中。流式细胞术允许对严重再生障碍性贫血中的糖基磷脂酰肌醇锚蛋白缺陷红细胞和中性粒细胞进行敏感和精确的测量。
设计和方法
我们对 207 例连续接受马抗胸腺细胞球蛋白加环孢素方案免疫抑制治疗的严重再生障碍性贫血患者进行了回顾性分析，这些患者在 2000 年至 2008 年间通过流式细胞术测量了阵发性睡眠性血红蛋白尿克隆。
结果
在 83 例（40%）患者的治疗前检测到糖基磷脂酰肌醇锚蛋白缺陷克隆的存在，克隆大小的中位数为 9.7%（四分位距 3.5-29）。在治疗前未检测到克隆的患者中，免疫抑制治疗后出现克隆的情况很少见，而在大多数治疗前有克隆的患者中，免疫抑制治疗后克隆大小通常会减少。然而，在 30 例患者中，观察到克隆大小增加。免疫抑制治疗后检测到阵发性睡眠性血红蛋白尿克隆的大多数患者乳酸脱氢酶没有升高，也没有发生溶血或血栓形成，并且不需要专门进行抗凝和/或依库珠单抗治疗。在需要治疗阵发性睡眠性血红蛋白尿症状和体征的 7 例患者中，所有患者乳酸脱氢酶升高，且克隆大小大于 50%。在所有患者中，18 例（8.6%）患者在任何给定的采样时间都有一个大于 50%的克隆。
结论
严重再生障碍性贫血中存在阵发性睡眠性血红蛋白尿克隆与较低的发病率和死亡率相关，很少需要采取特定措施来治疗临床阵发性睡眠性血红蛋白尿。
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