摘要
目的:总结环孢素(CsA)治疗儿童非重型再生障碍性贫血(NSAA)的长期效果,并探讨与CsA治疗NSAA起效和预后相关的早期指标。方法:回顾性分析2013年1月-2017年12月在华中科技大学同济医学院附属武汉儿童医院住院的36例NSAA患儿的病历资料,所有患儿在确诊后均接受CsA口服治疗,2周后检查CsA谷浓度以维持在200-250μg/L范围,评估时间点为口服CsA后3、6、12、18和24个月,评估项目为外周血细胞分类计数及网织红细胞百分比与计数。结果:36例患儿中男性16例,女性20例,中位年龄5.46(2.92-7.99)岁,中位随访时间28.00(10.00-38.25)个月。口服CsA后24个月临床治疗有效的累计病例数为21例。完全缓解4例,部分缓解17例,无效15例,总有效率58.33%,药物起效的中位时间为3.0(0.5-10.0)个月。与无效组比较,有效组(完全缓解和部分缓解)患儿表现为口服CsA后第3个月中性粒细胞和红细胞显著升高,第6个月血红蛋白、血小板、白细胞显著升高(P <0.05);口服CsA 3个月后有效组除2例患儿在3-12个月内进行成分输血外,其余19例患儿均不再需要成分输血支持治疗。结论:NSAA患儿口服CsA总体有效率为58.33%。治疗3个月后是否停止输注血液制品可作为疾病转归的转折点,治疗3个月时的中性粒细胞、红细胞和6个月时的血红蛋白、血小板、白细胞水平可作为评估疾病转归的指标。
Objective: To summarize the long-term efficacy of cyclosporine( CsA) in the treatment of non-severe aplastic anemia( NSAA) in children,and explore the early significant indicators. Methods: Data of 36 NSAA children in Department of Hematological Oncology,Wuhan Children’s Hospital,Tongji Medical College of Huazhong University of Science and Technology from January 2013 to December 2017 were analyzed retrospectively. All the children received oral CsA immunosuppressive therapy,and CsA trough concentration was checked to maintain at the rage of 200-250μg/L after 2 weeks. The evaluation time points were at 3,6,12,18 and 24 months,and assessment items were peripheral white blood cell differential count and reticulocyte’s percentage and count. Results: The 36 NSAA cases were composed of 16 males and 20 females,whose median age was 5. 46( 2. 92-7. 99) years old,and median follow-up time was 28. 00( 10. 00-38. 25) months. After taking oral CsA for 24 months,the number of cumulative effective cases was 21. There were 4 cases of complete remission( CR),17 cases of partial remission( PR),and 15 cases of non-remission( NR). The total effective rate was 58. 33%,and median effect-acting time of CsA was 3. 0( 0. 5-10. 0) months. Compared with ineffective group,neutrophil( NEU) and red blood cell( RBC) of effective group( CR+ PR) began to increase significantly at the 3 rd month,and hemoglobin( Hb),platelet( PLT) and white blood cell( WBC) increase significantly at the 6 th month after oral CsA administration( P < 0. 05). Except for 2 cases who received component transfusion within 3-12 months after taking oral CsA for 3 months in effective group,the others didnot need. Conclusion: The overall effective rate of oral CsA in children with NSAA was 58. 33%. Stopping blood transfusion after the 3 months of treatment may be considered as a turning point for disease outcomes,and levels of NEU,RBC at the 3 rd month and Hb,PLT,WBC at the 6 th month as indicators for predicting disease prognosis.
作者
卢文婕
张琳
杨李
王卓
聂应明
陈智
李晖
李建新
熊昊
LU Wen-Jie;ZHANG Lin;YANG Li;WANG Zhuo;NIE Ying-Ming;CHEN Zhi;LI Hui;LI Jian-Xin;XIONG Hao(Department of Hematological Oncology,Wuhan Children's Hospital,Tongji Medical College of Huazhong University of Science and Technology,Wuhan 430016,Hubei Province,China)
出处
《中国实验血液学杂志》
CAS
CSCD
北大核心
2021年第4期1257-1261,共5页
Journal of Experimental Hematology
基金
湖北省卫计委面上项目(WJ2017M195)
湖北省科技厅科研课题(2012FFB05302)
武汉市中青年医学骨干人才培养计划(武卫计(2014)77号)
武汉市卫计委科研课题(WX13B19)。
