The treatment of choice for aplastic anemia (AA) in children has been HLA-matched family donor (MFD) hematopoietic stem cell transplantation (HSCT). For those lacking MFD, immunosuppressive therapy (IST) consisting of horse antithymocyte globulin (ATG) and cyclosporine has been successful. The choices of second and third line treatments are more complex and debatable, especially in the situation of unavailability of horse ATG. IST with rabbit ATG seems to be less effective. Recently, improved survival of non-MFD HSCTs has been documented. The outcome of matched or mismatched unrelated donor, umbilical cord blood, or haploidentical family donor transplantations will be discussed in AA children after IST failure. Experimental approaches of upfront HSCT using non-MFDs will be briefly touched. In this review, a treatment guideline for children with AA from the Korean Society of Pediatric Hematology Oncology will be presented along with a brief review of literature on current clinical practices in Korea.
Keywords: Aplastic anemia; Children; Guidelines; Hematopoietic stem cell transplantation; Immunosuppressive treatment.