Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia

Kim, Inho; Lee, Dae Ho; Bang, Soo Mee; Byun, Jae Ho; Chang, Heung Moon; Lee, Moon Hee; Yoo, Young Jin; Ahn, Jin Seok; Lee, Jong Tae; Im, Seok Ah; Jung, Chul Won; Yang, Sung Hyun; Lee, Hong Bock; Suh, Cheolwon; Oh, Myung Don; Choe, Kang Won; Han, Kyou Sup; Park, Myoung Hee; Kim, Il Han; Ha, Sung Whan; Park, Charn Il; Jung, Kyung Hae; Park, Seonyang; Kim, Byoung Kook

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.