A phase II prospective study enrolling UBA1-mutated or wild type MDS patients with steroid-resistant or dependent autoinflammatory symptoms demonstrated that 75% (9/12) of patients with UBA1 mutations achieved complete or partial responses after 6 cycles of AZA treatment, with improvement in inflammation-related manifestations and sustainable steroid-sparing effects [56]. This evidence concerns the gene UBA1 and myelodysplastic syndrome.