One Hundred Cases of HLA-matched Sibling Allogeneic bone Marrow Transplantation in Children: Single Center Study

ABSTRACT

PURPOSE: We reviewed 100 cases of HLA-matched sibling allogeneic bone marrow transplantation(allo-BMT) in children and wish to share these results. MEHTODS One hundred children had undergone allo-BMT from HLA-identical siblings between Nov. 1983 and May 1998. There were 50 males and 50 females with a median age of 10 years and a median follow-up of 38 months. Out of 100 cases, 43 children were transplanted for severe aplastic anemia (SAA), 29 for acute myelogenous leukemia (AML), 18 for acute lymphocytic leukemia (ALL), 8 for chronic myelogenous leukemia (CML) and 2 for hemophagocytic lympho-histiocytosis (HLH). RESULTS: SAA The 5-year event free survival (EFS) of SAA was 91%. The types of events that occurred were 3 thrombotic thrombocytopenic purpura (TTP), 2 venoocclusive disease (VOD) and 1 rejection. AML In 25 of 29 cases, the 4-year EFS after allogeneic BMT in first remission was 71%. That of the TBI-based and Busulfan-based group was 44% and 77%, respectively. The most favorable results were observed in the Busulfan-based group in first remission with an EFS of 81% (n=18). The types of events that occurred were 4 TTP, 3 VOD, 2 rejections and 1 relapse. ALL Five-year EFS of children with complete remission (CR; n=14, 7 CR1, 7 CR2) was 81%. CML For the 6 children who received transplants while in the first chronic phase, the event free survival was 67%. HLH Both of the two children with HLH survived 9 months and 24 months after BMT, respectively. Acute GVHD (> or =Grade ll) was observed in 13 children. Chronic GVHD developed in 10 children; 8 cases were localized and 2 were extensive type. CONCLUSION: Allo-BMT can cure children with refractory stem cell disorders. The most important factor that influences survival after transplantation is interval between diagnosis and transplantation for patients with severe aplastic anemia and remission state at transplantation for patients with leu-