HK J Paediatr (new series) 2006;11:28-31

Abstract Fifteen children diagnosed of severe aplastic anaemia were treated with allogeneic haematopoietic stem cell transplantation (HSCT). The patients presented at the median age of 8.5 years, and 7 of 15 patients received HSCT within 2 months after diagnosis. The other patients had received immunosuppressive treatment but failed to response, they were then subsequently transplanted at 8-208 months after diagnosis. Ten patients had Human Leukocyte Antigen (HLA) identical sibling HSCT, whereas the other 5 patients received 1-3 antigen mismatched family donors (n=4) or unrelated donor transplant (n=1). Except two patients, all the other patients had successful engraftment of donor cells at median of 19 days. Those with successful engraftment did not have significant complications, and 5/13 patients developed graft versus host disease which responded to immunosuppressive treatment. Two patients without engraftment subsequently died of infection. The overall survival for the whole group was 86%, and it was 100 % for HLA identical or mismatched sibling transplant. At a median follow up of 7 years, the survivors were all in good health without late morbidity. In conclusion, HLA identical sibling HSCT achieved a high chance of cure and is the treatment of choice for children with severe aplastic anaemia. Key words Aplastic anaemia; Immunosuppressive therapy; Stem cell transplantatio