Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia / 대한혈액학회지

ABSTRACT

BACKGROUND: The outcome of hematopoietic stem cell transplantation (HSCT) for patients with severe aplastic anemia (SAA) in Seoul National University Hospital was analyzed retrospectively. METHODS: Between January, 1990 and March, 1999, 25 patients with SAA underwent HSCT. Their medical records were reviewed. Statistical analyses were done about survival and complication after HSCT. RESULTS: The median age of patients was 22 (range, 14~43) and male to female ratio was 18 7. Twenty two were HLA matched non- identical siblings. Three were one identical twin, one one-locus mismatched father and one HLA matched unrelated donor, respectively. Conditioning regimens were CY/TLI (cyclophosphamide, total lymphoid irradiation) for 18 patients, CY/ATG (CY, antithymocyte globulin) for 3, CY/ buffy (CY, unirradiated buffy- coat) for 2, CY/ ATG/TLI for 1, BU/CY (busulfan, CY) for 1. For prophylaxis of graft-versus-host disease (GVHD), cyclosporine and methotrexate were used in all patients except for identical twin. The median nucleated cell dose given to patients was 4.5x108/kg (range, 2.0~5.9). All evaluable patients achieved absolute neutrophil count of 500/microliter after median 17 days of HSCT (range, 12~27) and untransfused platelet count over 20,000/microliter after median 21 days of HSCT (range, 13~67). Six patients (24%, grade I 3, II 1, III 1, IV 1) developed acute GVHD and 8 (32%, limited 4, extensive 4) developed chronic GVHD. Hepatic venoocclusive disease (VOD) occurred in 2 patients (8%). Rejection occured in 4 patients (16 %), but among 22 allogeneic transplant recipients from HLA matched siblings, only one (5%) lost graft. After a median follow-up of 32 months (range 9~120 months), 5 year overall survival of all patients was 87%, and that of 22 allogeneic recipients from HLA matched sibling donors was 95%. Four patients (16%) died. Causes of death were VOD in one case, rejection with pneumonia one, acute GVHD one. One died from traffic accident in a cured state. CONCLUSION: Experiences from our center suggest that HSCT is an effective treatment for patients with severe aplastic anemia. Long- term survival is especially excellent for patients who have matched related donors.