Immunomodulation therapy for severe aplastic anemia--ALG versus ALG plus cyclosporin A.

Immunosuppressive treatment of aplastic anemia has been increasingly used as an alternative treatment to bone marrow transplantation. In this study, the additive effect of Cyclosporin A (CSA) (5mg/kg/day, at least 3 months) for maintenance of immunosuppression after antilymphocyte globulin (ALG) therapy (40mg/kg/day for 4 days) was compared to the previous ALG alone treatment (15mg/kg/day for 10 days). A high dose of methylprednisolone (20mg/kg/day for 5 days and 10mg/kg/day for 5 days) to the ALG group and a low dose of methylprednisolone (2mg/kg/day for 5 days) to the ALG plus CSA group were administered simultaneously. The results were as follows: 1) Sixteen (69.6%) out of twenty-three patients treated with ALG plus Cyclosporin A showed higher responses (CR: 48%, PR: 22%). On the contrary, nine out of nineteen in the ALG group showed lower responses (CR: 21%, PR: 26%). 2) Our data showed a tendency that male patients in age ranging from sixteen to thirty years showed an excellent response to ALG therapy (12/23: 52.2%). 3) The ALG plus CSA group revealed a faster response compared to the ALG alone group (15/16 within 6 months). 4) We speculate that ALG plus CSA therapy might be the treatment of choice for patients with a moderate degree of aplastic anemia. 5) Adding CSA to ALG increased the chance of infection, such as those with URI-like symptoms, but it did not affect the mortality rate. Our data suggest that the ALG plus CSA regimen may be a more useful therapeutic modality for patients with severe aplastic anemia who cannot be candidates for bone marrow transplantation and a randomized multicenter study is needed for confirmation of our preliminary study.

RCT Entities:   Population Interventions Outcomes