摘要
目的分析造血干细胞移植联合间充质干细胞输注对范可尼贫血(FA)患儿的治疗效果。方法通过分析1例.FA患儿外周血造血干细胞移植联合间充质干细胞输注治疗的临床资料,观察此种治疗方法对患儿的治疗效果及预后的影响。结果患儿于移植+13 d中性粒细胞植活,+15 d血小板植活,移植后供受者基因嵌合率为99.83%。移植后1.5个月血清CMV-DNA、EBV-DNA阳性,经免疫抑制剂减量及抗病毒治疗后转阴。移植后2个月出现皮肤GVHD(I度),未调整免疫抑制剂剂量,行间充质干细胞输注治疗4次后GVHD消失。移植后6个月,患儿出现血红蛋白水平下降,复查供受者基因嵌合率为57.8%,考虑出现移植物排斥,逐渐减停免疫抑制剂,供受者基因嵌合率复升至81.83%,血象恢复正常,无GVHD表现。现为移植后1.5年,患儿血象持续正常,供受者基因嵌合率维持在95%以上,FA相关基因检测持续转阴。结论造血干细胞移植是根治FA的重要治疗手段,健康供者骨髓间充质干细胞输注对于本例患儿造血干细胞植入、GVHD控制可能具有重要辅助作用。
Objective To study the effectiveness of hematopoietic stem cell transplantation （HSCT） combined with mesenchymal stem cells transfusion for Fanconi anemia （FA）. Methods A pediatric patient of 9 years old was studied, who had been diagnosed FA through the test of blood and bone marrow when she was 4 years old. In 2013, an HLA- identical sibling unrelated donor was found in Chinese Marrow Donor Programme（CDMP）. Peripheral blood stem cells were transfused after a reduced- intensity fludarabine-based conditioning regimen. Mesenchymal stem cells （1 x 106/kg） were given 4 hours earlier than peripheral blood stem cells transfusion for the purpose of regulating the immune function and promoting the engraftment of hematopoietic stem cells. Results The reconstitution time of peripheral blood granulocyte and platelet were respectively 13 days and 20 days after transplantation, Short tandem repeat（STR） DNA fingerprint test showed a 99.83% donor chimerism on day 20 after transplantation.Serum CMV-DNA and EBV-DNA showed positive 1.5 months after transplantation, and turned to be negative after antiviral treatments and reduced immune inhibitor. Grade-I acute GVHD occurred in 2 months after transplantation, then was controlled after two circles of mesenchymal stem cells transfusion without up-regulation of immune inhibitor. After 6 months of transplantation, peripheral blood STR showed a 57.8% donor chimerism, we gradually reduced immune inhibitor dose to zero. The chimerism rate returned to 81.83% after 4 months. In a 18-month follow up,donor chimerism is stable up to 95% and FA gene is negative. Conclusions HSCT is the radical cure for FA, and healthy donor mesenchymal stem cells may be useful and effective to regulating the immune function and promoting the engraftment of hematopoietic stem cells.
出处
《中国小儿血液与肿瘤杂志》
CAS
2015年第3期139-144,共6页
Journal of China Pediatric Blood and Cancer
基金
国家自然科学基金项目资助(项目批准号:31300747)
