【摘要】 目的探讨脐带间充质干细胞(umbilical cord mesenchymal stem cells,UCMSCs)治疗异基因造血干细胞移植(allogeneic hematopoietic stem cell transplantation,allo-HSCT)后儿童急性移植物抗宿主病(acute graft versus host disease,aGVHD)和出血性膀胱炎(hemorrhagic cystitis,HC)的疗效及安全性。方法 AlloHSCT后发生aGVHD 15例(合并HC 5例),难治性aGVHD的13例(合并HC 5例)对一线免疫抑制剂及CD25单抗治疗无效,联合静脉滴注UCMSCs治疗。UCMSCs的平均使用剂量为3.6(1.3~6.0)×106/kg,平均使用次数为5(2~21)次,中位随访时间47(36~73)个月。然后观察aGVHD和HC的恢复、再发情况及UCMSCs治疗相关不良反应。结果 13例难治性aGVHD联合UCMSCs治疗后8例完全缓解、5例部分缓解;5例HC患儿经水化、碱化治疗后效果欠佳,联合静脉滴注UCMSCs治疗后有效。未观察到UCMSCs治疗相关不良反应。结论 Allo-HSCT后联合UCMSCs治疗儿童aGVHD和HC安全有效。更多还原

【Abstract】 Objective To evaluate the efficacy and safety of umbilical cord mesenchymal stem cells( UCMSCs) in children with acute graft versus host disease( aGVHD) and hemorrhagic cystitis( HC) after allogeneic hematopoietic stem cell transplantation( allo-HSCT). Methods A total of 15 pediatric patients with refractory aGVHD or HC after allo-HSCT. Thirteen pediatric patients with aGVHD include 5 cases of HC with first-line immunosuppressor and CD25 monoclonal antibody. After failure treatment we gave combined intravenous infusion with UCMSCs. The mean dose of UCMSCs was 3. 6 × 10~6/kg [range( 1. 3 — 6. 0) × 10~6/kg] cells per kg bodyweight and the mean therapeutic times were 5( range 2 — 21).Then we observed the recovery,recurrence of aGVHD and HC and adverse reactions for 47 months( range 36 — 73). Results Thirteen cases after combined treatment with UCMSCs administration,and 8 patients had a complete response and 5 patients had a partial re-sponse. With a poor outcome after hydration and alkalization in 5 cases HC patients,symptoms disa-ppeared after combined intravenous infusion with UCMSCs. No patients had obvious side effect after infusion with UCMSCs. Conclusion UCMSCs are safe and effective for the combined treatment in children with aGVHD or HC after allo-HSCT.更多还原