Analysis of effect of allogeneic hematopoietic stem cell transplantation for children with myelodysplastic syndrome / 中华实用儿科临床杂志

ABSTRACT

Objective To analyze the clinic efficacy of allogeneic hematopoietic stem cell transplantation(alloHSCT) 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 received transplantation before Dec.2009 and 11 cases later.Results Up to the follow-up end point,6 cases died,and among 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 medical care have substantially improved HSCT outcome and reduced the treatment-related mortality.