摘要
目的分析异基因造血干细胞移植（allo．HSCT）治疗儿童骨髓增生异常综合征（MDS）的临床疗效，寻找可能影响预后的因素。方法回顾性分析18例2007年11月至2011年8月在上海交通大学医学院附属上海儿童医学中心接受allo—HSCT治疗的MDS患儿的长期随访资料，其中2009年12月前7例，之后11例。结果18例患儿中6例、死亡，其中3例复发，3例移植后发生治疗相关性死亡，2年总无病存活（DFS）率为（65．0±11．7）％。2009年12月前、后接受移植治疗的患儿2年DFS率有统计学差异[（28．6±17．1）％比（90．9±8．7）％，P=0．01]，采用白消安＋环磷酰胺（BUCy）作为预处理方案组患者的2年DFS率较其他方案组显著增高[（83．3±10．8）％比（25．0±20．4）％，P=0．035]。不同移植时间组患儿间仅预处理方案选择有统计学差异，其中2009年12月后组均采用BUCy方案（P=0．004）。结论allo—HSCT治疗能有效改善MDS患儿预后；BUCy方案作为儿童MDS预处理方案是合适的，移植相关经验的积累以及临床支持治疗技术和手段的进步对于降低治疗相关病死率、改善移植预后具有重要意义。
Objective To analyze the clinic efficacy of allogeneie hematopoietie stem cell transplantation（ allo- HSCT） for children with myelodysplastic syndromes（MDS） and to investigate the possible prognostic factors. Methods Eighteen children with MDS who underwent allo-HSCT at Shanghai Children＇s Medical Center Affiliated to Medical School of Shanghai Jiaotong University between Nov. 2007 and Aug. 2011 were retrospectively reviewed. Seven cases re- ceived transplantation before Dec. 2009 and 11 cases later. Results Up to the follow-up end point ,6 cases died, and a- mong them 3 cases died of relapse and 3 cases died of related treatment after transplantation. The 2-year disease free survival（DFS） rate of all patients was （65.0 ± 11.7 ）%. The 2-year DFS rate was significantly different between the group receiving transplantation before Dec. 2009 and the group receiving transplantation later [ （ 28.6 ± 17.1 ） % vs （90. 9 ± 8.7）% ,P = 0. 01 ]. Patients treated by regimen of busulfan/cyclophosphamide （BUCy） had a significant higher 2-year DFS rate compared with other treatment regimens [ （ 83.3 ± 10.8 ） % vs （ 25 ± 20.4 ） %, P = 0. 035 ]. The selected pretreatment regimen showed a significant difference between 2 groups in the different transplantation periods, and the pretreatment regimen of BUCy was more frequently used in both groups after Dec. 2009 （ P = 0. 004）. Conclusions Allo-HSCT is an effective treatment strategy for children with MDS and the pretreatment regimen of BUCy is suitable for those patients. The experience accumulation about transplantation and the improvement of supportive medi- cal care have substantially improved HSCT outcome and reduced the treatment-related mortality.
出处
《中华实用儿科临床杂志》
CAS
CSCD
北大核心
2013年第15期1150-1154,共5页
Chinese Journal of Applied Clinical Pediatrics
关键词
骨髓增生异常综合征
异基因造血干细胞移植
复发
治疗相关病死率
JD童
Myelodysplastie syndrome
Allogeneie hematopoietic stem cell transplantation
Relapse
Treat- ment-related mortality
Child
