Good outcome of haploidentical hematopoietic SCT as a salvage therapy in children and adolescents with acquired severe aplastic anemia.

Haploidentical hematopoietic SCT (haplo-HSCT) is to be established in patients with acquired severe aplastic anemia (SAA) refractory to immunosuppressive therapy and lacking HLA-matched related or unrelated donors. Graft failure (GF) and GVHD have been major obstacles to HSCT. A total of 17 children and adolescents with SAA underwent haplo-HSCT in our center. The conditioning regimen consisted of BU, fludarabine, CY and anti-thymocyte globulin. All patients received cyclosporine, short-term MTX, mycophenolate mofetil and basiliximab for GVHD prophylaxis. Mesenchymal stem cells derived from unrelated umbilical cord were infused on day 1. Neutrophil engraftment was achieved in all 17 patients in a median time of 16 days (range 9-25 days). The median time of platelet engraftment was 22 days (range 9-95 days) in 16 patients. The cumulative incidence (CI) of II-IV acute GVHD (aGVHD) at day +100 was 30.53±11.12% and III-IV aGVHD occurred in only one patient. The CI of chronic GVHD was 21.25±13.31%. Secondary GF with autologous hematopoiesis recovery occurred in one patient. The OS was 71.60±17.00% at a median follow-up of 362 (36-1321) days. These limited promising data suggest that haplo-HSCT is feasible as a salvage therapy for children and adolescents with refractory SAA who lack matched donors.