Methods: Sixty-five patients (34 males/31 females) reported to the registry of the European Working Group (EWOG) of MDS and treated with HSCT for GATA2-related MDS were diagnosed with RCC (36), MDS-EB (22), MDS-EBt (6) or MDR-AML (1) at a median age of 12.8 yrs. The gene discussed is GATA2; the disease is acute myeloid leukemia.