To 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.

Retrospective 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.

All 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.

It is safe and effective to treat children's SAA with hi-HSCT combined with hUC-MSC infusion and is worthy of further attention.