Down syndrome and AML: Where do we go from here?

Abstract

It is well documented that young children with Down syndrome (DS) have both a 500-fold increased incidence of acute myeloid leukemia (ML-DS) and a decreased tolerance of intensive chemotherapy. In this issue of Blood, Uffmann et al present the results of a large, multicentered, international, nonrandomized trial reducing the etoposide exposure while preserving the excellent outcomes reported in previous trials.1 This trial builds on international experience demonstrating that most young children with ML-DS may be cured with less intensive therapy, and confirms that there remains a significant subset of patients for whom we have limited therapeutic options.