The HSCT data presented here, in particular the highly similar outcome in GATAmut as compared to GATAwt patients with respect to OS, DFS, NRM and relapse, support the hypothesis that the currently recommended EWOG-MDS algorithm for therapy of pediatric MDS can also be applied to children with GATA2 deficiency. This evidence concerns the gene GATA2 and hyperinsulinemic hypoglycemia, familial, 4.