儿童难治性血细胞减少的临床特征分析

安文彬; 张培红; 任媛媛; 郭晔; 王书春; 陈晓娟; 杨文钰; 常丽贤; 竺晓凡

doi:10.7499/j.issn.1008-8830.2015.01.004

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中国当代儿科杂志 ›› 2015, Vol. 17 ›› Issue (1) : 15-21. DOI: 10.7499/j.issn.1008-8830.2015.01.004

论著·临床研究

儿童难治性血细胞减少的临床特征分析

安文彬¹, 张培红², 任媛媛¹, 郭晔¹, 王书春¹, 陈晓娟¹, 杨文钰¹, 常丽贤¹, 竺晓凡¹

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Clinical features of childhood refractory cytopenia

AN Wen-Bin¹, ZHANG Pei-Hong², REN Yuan-Yuan¹, GUO Ye¹, WANG Shu-Chun¹, CHEN Xiao-Juan¹, YANG Wen-Yu¹, CHANG Li-Xian¹, ZHU Xiao-Fan¹

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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1 420 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 1 420 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.

Abstract

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-classified using the criteria proposed in the 2008 edition of the World Health Organization classification 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.

导出引用

安文彬, 张培红, 任媛媛, 郭晔, 王书春, 陈晓娟, 杨文钰, 常丽贤, 竺晓凡. 儿童难治性血细胞减少的临床特征分析[J]. 中国当代儿科杂志. 2015, 17(1): 15-21 https://doi.org/10.7499/j.issn.1008-8830.2015.01.004

AN Wen-Bin, ZHANG Pei-Hong, REN Yuan-Yuan, GUO Ye, WANG Shu-Chun, CHEN Xiao-Juan, YANG Wen-Yu, CHANG Li-Xian, ZHU Xiao-Fan. Clinical features of childhood refractory cytopenia[J]. Chinese Journal of Contemporary Pediatrics. 2015, 17(1): 15-21 https://doi.org/10.7499/j.issn.1008-8830.2015.01.004

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