Allogeneic Bone Marrow Transplantation Experience for Children with Severe Aplastic Anemia and Refractory Leukemia

ABSTRACT

PURPOSE: We reviewed the result of allogeneic bone marrow transplantation(BMT) from HLA-identical sibling donors in children with refractory stem cell disorder along with future implication. METHODS: Forty-two children with refractory stem cell disorder received BMT from HLA-identical sibling donors between Nov. 1983 and Feb. 1995. Out of 42 children, 23 cases were severe aplastic anemia(SAA) and 19 cases were refractory leukemias. There were 20 male and 22 female with median age of 13 years (range, 2-17) and median follow-up of 36 months (range, 4-139 months). RESULTS: 1) The overall survival rate of all patients was 73.8%. The survival rate for SAA cases was 87.0%, while that for leukemia was 57.9%. 2) Acute GVHD(> or = grade II) was observed in 16.7% of all patients, 8.7% of SAA patients and 26.3% of leukemia patients, respectively. Chronic GVHD developed in 9.5% of all patients, 4.8% of limited type and 4.8% of extended type. No death was directly attributable to GVHD. 3) The causes of death after allogeneic BMT were graft rejection(7.1%), relapse of leukemia(7.1%), thrombotic thrombocytopenic purpura(4.8%), veno-occlusive disease, sepsis and CMV pneumonia respectively 2.4%. 4) The most common complication except death after allogeneic BMT was herpes zoster(26.2%). The other complications were hemorrhagic cystitis(7.1%), bronchiolitis obliterans and measles respectively 2.4%. CONCLUSIONS: We confirmed that allogeneic BMT is the curable treatment for children with refractory stem cell disorder. The most important factors that influence the result of transplantation are interval between diagnosis and transplantation in severe aplastic anemia and remission state at transplantation in leukemia.