混合移植治疗儿童血液病疗效观察

本研究探讨HLA不全相合骨髓造血干细胞及外周造血干细胞、加入第三方脐血造血干细胞混合移植治疗小儿血液病的疗效。对2012年8月至12月我院5例难治性血液病患儿进行异基因造血干细胞移植，移植方式为HLA不全相合骨髓造血干细胞、外周血造血干细胞及脐血造血干细胞混合移植，观察混合移植中的第三方脐血干细胞对于移植后造血重建时间、STR嵌合度、GVHD发生情况及移植相关近期并发症的作用。结果表明，5例患儿移植后均获得造血重建，ANC〉0．5×10^9／L的中位时间是移植后11d，Pit〉20×10^9／L的中位时间移植后10d；外周血STR-PCR嵌合度检测显示，于移植后30d均达到稳定嵌合；5例患儿出现轻至中度GVHD症状，表现为Ⅰ-Ⅱ皮肤GVHD，其中2例患儿发生腹泻，出现Ⅰ-Ⅱ肠道GVHD，5例患儿均无肝功能损害。随访至2013年4月30日，中位随访时间137d（130d-250d），1例移植后131d死于严重出血性膀胱炎及多部位感染，其余4例均无病生存至今，存活时间分别为130、137、193、250d，中位生存时间134d。结论：HLA不全相合骨髓造血干细胞、外周血造血干细胞、第三方脐血造血干细胞混合移植对提高小儿血液病的存活率、延长存活时间有一定疗效。

Therapeutic Efficacy of Mixed Hematopoietic Stem Cell Trans- plantation for Pediatric Hematologic Diseases

This study was purposed to explore the effectiveness of mixed transplantation of HLA mismatched bone marrow hematopoietic stem cells（ HSC）, peripheral blood HSC and umbilical cord blood HSC for treatment of pediatric blood diseases. From August 2012 to December 2012, five children with refractory hematological diseases in our hospital received allogeneic hematopoietic stem cell transplantation. The mixed grafts consisting of HLA-mismatched bone mar- row HSC, peripheral blood HSC and umbilical cord blood HSC were used to observe the effects of umbilical cord blood HSC on the time of hematopoietic reconstraction of bone marrow, STR chimeric degrees, incidence of GVHD. and early transplant-assosiated comlications. The results showed that all 5 children patients were grafted successfully with the medi- an grafted time of 11 d for ANC 〉0.5 ×109/L and 10 d for Plt 〉20 × 109/L, respectively. On day 30, the STR-PCR test of peripheral blood showed a stable complete chimera. Five cases suffered from mild to moderate symptoms of GVHD, showing with Ⅰ - Ⅱ grade of skin GVHD and in which two cases suffered from diarrhea, showing Ⅰ - Ⅱ grade of intestinal GVHD. All the 5 patients had no liver function damage. One patient died of severe hemorrhagic cysti- tis and multi-site infection, and the remaining four cases survived so far on the current median follow-up time of 137 d （ 130 d - 250 d）. It is concluded that transplantation of the mixed HLA mismatched bone marrow HSC, peripheral blood HSC, with third-party cord blood HSC can increase the survival rate for pediatric patients with blood disease.