Immunosuppressive treatment of aplastic anemia in Chinese children with antithymocyte globulin and cyclosporine.

Fifty-one children with aplastic anemia (AA) from 1993 to 2004 in the authors' institution were treated by 3 therapies: 11 patients in group 1 received the SSL-6 protocol; 16 patients in group 2 had CsA alone, where the dose of CsA began from 9-12 mg/kg in the initial 2 weeks and tapered off to 5 mg/kg later; 24 patients in group 3 were treated combining rabbit ATG (Pasteur, Merieux) 2.5 mg/kg for 5 days with CsA, which was the same dose as in group 2. The response was 27, 50, and 79%, respectively. The statistical analysis showed that the protocol of intensive immunosuppressive treatment (IST) was the most effective one and CsA was better than that of SSL-6. None of our patients developed clone diseases although the follow-up was as long as more than to 9 years. The data suggest that children with AA should receive IST as first-line therapy in developing countries. Hematopoietic stem cell transplantation (HSCT) is effective treatment for patients with aplastic anemia (AA). However, HSCT is not widely used in China for economic reasons and lack of donors. Immunosuppressive therapy (IST) is now the mainstay treatment for AA. To evaluate the effect of immunosuppressive therapy, combining antithymocyte globulin (ATG) with cyclosporine (CsA), a retrospective study on 51 children with AA from January 1993 to December 2004 treated in the authors' department was performed.