Long-term observation of pediatric aplastic anemia

Fifty-nine verified cases of acquired aplastic anemia (AA), diagnosed at the Pediatric Department of the National Taiwan University Hospital from 1977 to 1987, were reviewed and analyzed. The demographic features showed a high relative incidence (acute myelogenous leukemia/AA ratio, 2.2/1), a high percentage of non-severe AA (39%) and a high association with hepatitis (20.8%). No evidence of hepatitis A, B or C virus infection was found in five cases of hepatitis-associated AA. No sex preponderance was noted in this pediatric series. The 10-year projected survival rate of the total series approached 55%. The crude two-year-survival and two-year-transfusion-free-survival rates were 59% and 44%, respectively, in the conservative therapy group treated with androgens and steroids; 36% and 32%, respectively, in patients with severe AA in the conservative therapy group; and 73% and 64%, respectively, in the aggressive therapy group treated with cyclosporin, anti-lymphocyte globulin or bone marrow transplant. The major causes of death were hemorrhage (44%) and infection (56%) in the conservative therapy group; but in the aggressive therapy group, two out of three deaths were related to therapeutic complications. Multivariate analysis of prognostic factors revealed that severity and treatment modality were independent risk factors. Only two out of 31 patients who survived more than two years (long-term survivors) experienced late mortalities. At two, five, seven and 10 years after diagnosis, 61%, 55%, 41% and 40% of the long-term survivors had inadequate hematopoietic recovery.