Acquired aplastic anemia in childhood

In comparison to past decades, children who have acquired aplastic anemia (AA) enjoy excellent overall survival that reflects improvements in supportive care, more accurate exclusion of children who have alternate diagnoses, and advances in transplantation and immunosuppressive therapy (IST). Matched sibling-donor hematopoietic stem cell transplants (HSCT) routinely provide long-term survival in the range of 90%, and 75% of patients respond to IST. In this latter group, the barriers to overall and complication-free survival include recurrence of AA, clonal evolution with transformation to myelodysplasia/acute myelogenous leukemia, and therapy-related toxicities. Improvements in predicting responses to IST, in alternative-donor HSCT, and in rationalizing therapy by understanding the pathophysiology in individual patients are likely to improve short- and long-term outcomes for these children.