Treatments of Severe Aplastic Anemia in Children

Abstract

We carried out a retrospective analysis of the outcome of treatment in patients with severe aplastic anemia who attended the Department of Pediatrics, Siriraj Hospital, during 1972- 1998. There were 31 patients, 17 boys and 14 girls, by Camitta’s criteria for severe aplastic anemia. All of them were idiopathic. Twenty patients were treated conventionally with steroid and androgen compounds, 5 with immunosuppressive therapy and 6 with bone marrow transplantation. In the conventional treatment group, after 1 year to 19 years of follow up, 64.7% achieved complete response, 11.8% achieved partial response and 23.5% died. The response rate in the immunosuppressive therapy group was only 40% after 3 months to 2 years of follow up. In the bone marrow transplantation group, bone marrow engraftment was achieved in all cases (means 29.8, range 19 - 42 days), and the patients yielded the highest complete response rate (100%). However, 1 case relapsed after complete response for 1 year, but he was successfully cured after second transplantation. The overall survival rate and cure rate were also 100% after 1 year and a half to 10 years of follow up. Bone marrow transplantation, when compared to other treatments, resulted in the highest response or cure rate with the shortest treatment duration. The only disadvantages of bone marrow transplantation are the high cost and the limited availability of HLA compatible donors.