造血干细胞移植治疗儿童急性重型再生障碍性贫血的效果

王彬+吴润晖++庞一琳++朱光华++周翾++杨骏+++李斯丹++秦茂权

[摘要] 目的 探讨造血干细胞移植（HSCT）治疗儿童急性重型再生障碍性贫血的效果，评价其安全性及预后。 方法 回顾性分析首都医科大学附属北京儿童医院血液科2006年1月～2015年12月采用HSCT治疗51例急性重型再生障碍性贫血患儿的临床资料，评价其植入情况、并发症及移植疗效。 结果 98%患者获得造血重建。中性粒细胞≥0.5×109/L的中位时间为11 d；血小板≥20×109/L的中位时间为15 d。10例合并巨细胞病毒血症，2例合并出血性膀胱炎，7例发生Ⅰ～Ⅱ度急性移植物抗宿主病，2例发生Ⅲ度急性移植物抗宿主病。46例患者存活，3年存活率Allo-HSCT、Haplo-HSCT、MUD-HSCT分别是100%、89%、75%，无感染<5岁的极重型再生障碍性贫血任何移植成功率均达100%。 结论 HSCT治疗儿童急性重型再生障碍性贫血的方法有效，并发症少，生存率高。

[关键词] 重型再生障碍性贫血；造血干细胞移植；儿童

[中图分类号] R556 [文献标识码] A [文章编号] 1673-7210（2017）07（b）-0097-04

Stem cell transplantation for children with acquired severe aplastic anaemia

WANG Bin1 WU Runhui1 PANG Yilin2 ZHU Guanghua1 ZHOU Xuan1 YANG Jun1 LI Sidan1 QIN Maoquan1▲

1.Hematology Oncology Center， Beijing Children′s Hospital Affiliated to Capital Medical University Beijing Key Laboratory of Hematological Diseases and Tumor Molecular Classification National Key Discipline of Pediatrics， Beijing 100045， China； 2.Department of Emergency， Beijing Children's Hospital Affiliated to Capital Medical University， Beijing 100045， China

[Abstract] Objective To explore the effect of hematopoietic stem cell transplantation （HSCT） in the treatment of severe aplastic anemia， and evaluate its safety and prognosis. Methods Clinical data of 51 children with severe aplastic anaemia received HSCT in Hematology Department of Beijing Children′s Hospital Affiliated to Capital Medical University from January 2006 to December 2015 were retrospectively analyzed. The engraftment， complications and curative effect were evaluated. Results 98 percent of patients had rapid engraftment. The median time of neutrophil exceeded 0.5×109/L and platelets recovery exceeded 20×109/L were 11 and 15 days for all transplantation patients. In this research， 10 patients were diagnosed with CMV viremia. 2 patients developed hemorrhagic cystitis. 7 cases had grade Ⅰ-Ⅱ aGVHD and 2 cases had grade Ⅲ aGVHD. 46 patients were still alive. The estimated 3-year OS of the Allo-HSCT， Haplo-HSCT and MUD-HSCT group was 100%， 89% and 75% respectively. All children less than 5 years of age （without infection） were transplanted successfully. Conclusion HSCT is effective in the treatment of acute severe aplastic anemia in children， with fewer complications and high survival rates

[Key words] Severe aplastic anemia； Hematopoietic stem cell transplantation； Children

再生障礙性贫血（aplastic anemia，AA）是由某种或多种复合因素引起的原发性或继发性的骨髓造血功能衰竭，因造血增生不良和外周血全血细胞减少，导致贫血、出血和反复感染为特征的一组综合征[1]，根据患者的血象及骨髓象，依据病情的严重程度，分为非重型再生障碍性贫血（non-severe aplastic anemia，NSAA）和重型再生障碍性贫血（severe aplastic anemia，SAA），其中SAA中的外周血中性粒细胞绝对值（absolute neutrophil count，ANC）<2×109/L时，又称为极重型再生障碍性贫血（very severe aplastic anemia，VSAA）。NSAA通常起病及进展缓慢，病情较SAA轻，治疗多以控制感染、输血、促造血治疗等支持治疗为主，多数可缓解甚至治愈，少数进展为SAA[2]。而SAA以发病急，病情重，常伴有严重感染、出血及贫血为特点，病死率高，输血、促造血等支持治疗后通常病情难以控制，目前治疗主要以免……