Department of Internal Medicine, Seoul National University Hospital, College of Medicine, 28 Yongon-dong, Chongno-gu, Seoul, 110-744, Republic of Korea.
Int J Hematol. 2010 Jun;91(5):770-5. doi: 10.1007/s12185-010-0601-1. Epub 2010 Jun 5.
The clinical course of non-severe aplastic anemia is variable, and risk factors related to disease progression are not well known. We reviewed clinical and laboratory data of the patients who were diagnosed with non-severe aplastic anemia from 1997 to 2007 at Seoul National University Hospital and analyzed the clinical course and outcomes in these patients. We defined non-severe aplastic anemia as hypocellular marrow with cytopenia in the peripheral blood, which does not meet the criteria for severe aplastic anemia (at least two of the following: ANC < 500/microl, platelet < 20,000/microl or reticulocyte < 20,000/microl). Among a total of 96 patients, 53 (55.2%) were male and the median age was 37.6 years old. As much as 41.7% (40) of the patients were initially asymptomatic. Sixty-two patients who were treated with oxymetholone, ATG/ALG, cyclosporin or other agents after initial diagnosis showed significantly lower levels of initial hemoglobin, red blood cell count and platelet count than those who did not receive any treatment. During the follow-up period, 18 patients progressed to severe aplastic anemia. Their median age was 29.9 years and the median progression time was 18 months. Initial white blood cell count and absolute neutrophil count in the evolution group tended to be lower than in the other group. The patients whose thrombocytopenia did not respond to treatment showed markedly higher frequency of progression to severe aplastic anemia. Treatment itself and responsiveness in reticulocyte and absolute neutrophil count were not correlated with their clinical courses. Sixteen patients showed overall improvement, whereas three patients developed secondary hematologic disease, acute myeloid leukemia, myelodysplastic syndrome and paroxysmal nocturnal hemoglobinuria. Non-severe aplastic anemia has a relatively indolent and mild clinical course. However, 18.8% of the study population progressed to severe disease. White blood cell and absolute neutrophil count at diagnosis and treatment responsiveness of thrombocytopenia were associated with disease progression. Careful monitoring and early management are needed for patients at risk.
非重型再生障碍性贫血的临床病程多变，与疾病进展相关的危险因素尚不清楚。我们回顾了 1997 年至 2007 年在首尔国立大学医院诊断为非重型再生障碍性贫血的患者的临床和实验室数据，并分析了这些患者的临床病程和结局。我们将非重型再生障碍性贫血定义为骨髓细胞减少伴外周血血细胞减少，不符合重型再生障碍性贫血标准（至少以下两项：ANC<500/μl、血小板<20,000/μl 或网织红细胞<20,000/μl）。在总共 96 例患者中，53 例（55.2%）为男性，中位年龄为 37.6 岁。多达 41.7%（40 例）的患者最初无症状。62 例患者在初始诊断后接受了氧甲氢龙、ATG/ALG、环孢素或其他药物治疗，其初始血红蛋白、红细胞计数和血小板计数明显低于未接受任何治疗的患者。在随访期间，18 例患者进展为重型再生障碍性贫血。他们的中位年龄为 29.9 岁，中位进展时间为 18 个月。进展组的初始白细胞计数和绝对中性粒细胞计数往往低于其他组。血小板治疗无反应的患者进展为重型再生障碍性贫血的频率明显更高。治疗本身以及网织红细胞和绝对中性粒细胞计数的反应性与他们的临床病程无关。16 例患者总体改善，而 3 例患者发生继发性血液系统疾病、急性髓系白血病、骨髓增生异常综合征和阵发性夜间血红蛋白尿。非重型再生障碍性贫血的临床病程相对缓慢和轻微。然而，18.8%的研究人群进展为严重疾病。诊断时的白细胞和绝对中性粒细胞计数以及血小板治疗反应性与疾病进展相关。需要对高危患者进行密切监测和早期管理。
