CYCLOSPORINE-A IN THE TREATMENT OF SEVERE APLASTIC-ANEMIA IN CHILDREN

Purpose: To evaluate the response of children with severe aplastic ane mia (SAA) treated with cyclosporin A (CSA). Patients and Methods: Retr ospective analysis was done on the data of 15 patients with SAA who we re treated with cyclosporin with or without additional immunosuppressi ve therapy. Results: Eight of 15 patients responded. The median time t o recovery was as follows: 32 days for absolute neutrophil count >1.0x 10(9)/l; 115 days for platlet count >75x10(9)/l; and 154 days for hemo globin >10g/dl. Three patients were successfully weaned off CSA. Four of 7 patients, whose therapy was discontinued, required retreatment wi th CSA. Seven of 8 responders are transfusion-independent with a media n follow-up of 51 months. Only 3 to 7 nonresponders are alive: 1 after bone marrow transplantion and 2 require supportive care. Conclusions: CSA offers a therapeutic option for pediatric patients with SAA who a re awaiting bone marrow transplantation or do not have a donor. Althou gh the treatment is not curative, and a significant number of patients relapse after discontinuation of the drug, some patients can achieve remission.