The Mortality of Acquired Aplastic Anemia in Children

The course of 58 children under age 16 yr with verified acquired aplastic anemia, treated at the Children’s Hospital Medical Center of Boston, 1958-70, has been reviewed. All but one child received androgen, corticosteroid, and supportive therapy. Forty-one of the children (71%) have died, 15 (26%) are alive, and 2 (3%) have been lost to follow-up. Deceased patients had a higher proportion of lymphocytes (p <0.01) and a relative deficiency of hematopoietic precursor cells on initial bone marrow examination. They did not differ significantly from survivors with regard to age, sex, or fetal hemoglobin concentration, and nearly all patients had intense depression of the initial peripheral blood counts. The median survival for the group was less than 6 mo; all 11 new patients hospitalized over the last 5 yr died within 6 mo of starting treatment with oxymetholone and other androgen preparations. Evaluation of treatment for acquired aplastic anemia in children is difficult because of the variable natural history of the disorder. Our findings suggest that androgen therapy often fails to alter the short lethal course of the disease in children requiring hospitalization for complications of severe pancytopenia.