摘要
目的：研究儿童难治性血细胞减少（RCC）的临床特征。方法回顾性分析1990年1月到2013年6月1420例初诊为非重型再生障碍性贫血儿童（0-14岁）的临床资料，重新评估患儿骨髓细胞形态和组织病理，按照2008版WHO造血与淋巴组织肿瘤中RCC诊断标准重新进行诊断分型。患儿临床转归通过每3-6月门诊就诊随访获得。结果1420例患儿中，152例（10.7%）诊断为RCC。与非重型再生障碍性贫血患儿比较，RCC患儿发病时血红蛋白较低，胎儿血红蛋白比例增高。RCC患儿诊断时异常核型者比例为21.5%。中位随访时间为36个月（范围1-283个月）。RCC患儿对环孢素和雄激素治疗的完全有效、部分有效和无效率分别为19.0%、26.7%、54.3%。RCC患儿5年和10年的预期生存率分别为87.9%和72.4%；5年和10年的预期克隆演变率分别为15.3%和20.0%；2年预期新发染色体核型异常率为3.6%；5年和10年预期白血病转化率为10.0%和20.0%。结论 RCC具有成人骨髓增生异常综合征的基本特征。RCC患儿预后差，向白血病转化风险高，环孢素治疗有效率较低。
Objective To study the clinical features of patients with refractory cytopenia of childhood （RCC）. Methods The clinical data of 1 420 children （0-14 years old） with an initial diagnosis of non-severe aplastic anemia between January 1990 and June 2013 were retrospectively analyzed. Bone marrow cell morphology and histopathology were re-evaluated, and the patients were re-classiifed using the criteria proposed in the 2008 edition of the World Health Organization classiifcation of RCC in hematopoietic and lymphoid tumor tissues. The clinical outcomes were followed up every 3-6 months. Results Among all the 1 420 cases, 152 （10.7%） were reassessed as RCC. Patients with RCC had a lower level of hemoglobin and a higher percentage of fetal hemoglobin than those with non-severe aplastic anemia. Of the patients with RCC, 21.5%showed abnormal karyotypes at diagnosis. The median follow-up period for all patients was 36 months （ranging from 1 to 283 months）. The rates of complete response, partial response, and no response to cyclosporine and androgen treatment in RCC patients were 19.0%, 26.7%, and 54.3%, respectively. The 5-and 10-year prospective overall survival rates of RCC patients were 87.9%and 72.4%, respectively. The 5-and 10-year prospective clonal evolution rates were 15.3% and 20.0%, respectively. The 2-year prospective incidence of newly diagnosed karyotype abnormality after the initial diagnosis was 3.6%. The 5-and 10-year prospective leukemia transformation rates were 10.0%and 20.0%, respectively. Conclusions RCC shows clinical features similar to adult myelodysplastic syndrome. Children with RCC have a poor prognosis, an increased risk of transformation to leukemia, and a low response rate to cyclosporine treatment.
出处
《中国当代儿科杂志》
CAS
CSCD
北大核心
2015年第1期15-21,共7页
Chinese Journal of Contemporary Pediatrics
基金
2010-2012卫生部部属(管)医院学科重点项目
