单倍体相合造血干细胞移植联合脐带间充质干细胞输注治疗儿童重型再生障碍性贫血的临床观察及安全性分析

目的观察单倍体相合造血干细胞移植（hi-HSCT）联合脐带间充质干细胞（hUC-MSC）输注治疗儿童重型再生障碍性贫血（SAA）的临床效果及安全性。 方法整理海军总医院儿科2010年2月至2014年1月收治的11例接受hi-HSCT联合hUC-MSC输注治疗的SAA患儿的临床资料，进行回顾性分析。对其治疗情况、并发症发生情况及生存情况进行观察，总结该治疗方案的临床效果与安全性，以及治疗体会。 结果患儿全部获得造血重建，移植后1个月复查嵌合体均为70% ~ 100%供者嵌合。白细胞植入时间8 ~ 21 d，中位时间为12 d；血小板植入时间10 ~ 24 d，中位时间为15 d。11例患儿中，2例发生Ⅰ度急性移植物抗宿主病（GVHD），1例发生Ⅲ度急性GVHD，均经相关治疗后好转；1例发生局限性慢性GVHD，经相关治疗后好转；2例发生广泛性慢性GVHD，发生率为18.18%。患儿移植期间均发生不同程度的恶心、呕吐、纳差和发热等症状，给予对症支持治疗后好转。8例（72.73%）发生口腔黏膜炎，2例（18.18%）发生肺部感染，9例（81.82%）发生病毒感染，2例（18.18%）发生腹泻，均经综合治疗后好转。11例患儿随访时间12 ~ 29个月，中位随访时间16个月，截止末次随访时1例因广泛性慢性GVHD接受持续治疗，2例接受免疫抑制剂减量治疗，其余4例均停用免疫抑制剂；1例患儿因家属自行停用环孢素A发生排异死亡。 结论hi-HSCT联合hUC-MSC输注治疗儿童SAA具有良好临床疗效，值得进一步关注。

Clinical observation and safety analysis of haploidentical hematopoietic stem cell transplantation combined with umbilical cord mesenchyme stem cell infusion in the treatment of children with severe aplastic anemia

ObjectiveTo observe the clinical efficacy and safety of children with severe aplastic anemia (SAA) with haploidentical hematopoietic stem cell transplantation (hi-HSCT) combined with human umbilical cord mesenchymal stem cells (hUC-MSC) infusion. MethodsRetrospective analysis of 11 children with SAA was performed in our hospital in January 2014 ~ February 2010 treated with hi-HSCT combined with hUC-MSC infusion. The treatment situation, the occurrence of complications and the survival situation was observed, and the clinical effect and safety of the treatment plan, as well as the experience of treatment was summarized. ResultsAll the children received hematopoietic reconstitution, and the chimerism was 70%~ 100% 1 months after transplantation. The time of leukocyte implantation was 8 ~ 21 d (median time, 12 d), and the time of platelet implantation was 10 ~ 24 d (median time, 15 d). Among the 11 patients, 2 had grade 1 acute GVHD, 1 had grade III acute GVHD, and all of them were improved after related treatment; 1 had localized chronic GVHD, and was improved after related treatment; 2 had widespread chronic GVHD, and the incidence rate was 18.18%. The patients during transplantation had different degrees of nausea, vomiting, anorexia, fever and other symptoms, and were improved after symptomatic support treatment. 8 patients (72.73%) with oral mucositis (18.18%), 2 with pulmonary infection, 9 (81.82%) with virus infection and 2 (18.18%) with diarrhea were improved after comprehensive treatment. 11 patients were followed up for 12 ~ 29 months, and the median follow-up time was 16 months. 1 patient received extra treatment because of extensive chronic GVHD, 2 received continuous immunosuppression reduction treatment, and the remaining 4 were discontinued immunosuppressants; 1 patient died due to family members' withdrawal of cyclosporine arbitrarily at the end of the follow-up. ConclusionIt is safe and effective to treat children's SAA with hi-HSCT combined with hUC-MSC infusion and is worthy of further attention.