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.
Article PDF
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Werner, E., Stout, R., Valdez, L. et al. FAVORABLE PROGNOSIS FOR CHILDREN WITH APLASTIC ANEMIA TREATED WITH IMMUNOSUPPRESSIVE THERAPY. Pediatr Res 21 (Suppl 4), 307 (1987). https://doi.org/10.1203/00006450-198704010-00841
Issue Date:
DOI: https://doi.org/10.1203/00006450-198704010-00841