FAVORABLE PROGNOSIS FOR CHILDREN WITH APLASTIC ANEMIA TREATED WITH IMMUNOSUPPRESSIVE THERAPY

Abstract

Survival rates for children with aplastic anemia (AA) who do not receive HLA-matched bone marrow transplantation (BMT) are less than 50%. In the past 5 years we have treated 9 patients (pts) with AA with immunosuppressive therapy (ImmRx); 8 pts are alive and do not require transfusion (Tx) with a median follow-up of 14.5 months (range 5-62). Seven pts had severe AA and 2 had moderate AA. AA was associated with hepatitis in 2 pts, benzene in 1 pt, arthritis in 1 pt and was idiopathic in 5 pts. All pts received anti-thymocyte globulin (ATG) 15 mg/kg/day for 14 consecutive days followed by 7 additional doses over the next 14 days. All pts received methylprednisolone or its equivalent at 1-2 mg/kg/day during the ATG to combat serum sickness. Two pts also received 1 haploidentical bone marrow infusion and oral androgens, 1 additional pt received oral androgens. Six pts (5 severe) had a good response to ATG and are Tx free. In one pt, persistent thrombocytopenia responded to cimetidine therapy. Of the 3 pts who failed ATG, 2 are Tx free after treatment with cyclosportne A. During the same time, 6 pts with severe AA had BMT with a complete response. The response to BMT was significantly faster than to ATG. Red cell Tx was required for 34 days post BMT vs. 66 days post ATG (p<0.05). In conclusion, although BMT is better than ImmRx in children with AA, when a suitable donor is unavailable, ImmRx may result in a favorable outcome.