UBA1 and myelodysplastic syndrome: This biological heterogeneity translates directly into disparate treatment outcomes, for example; high-dose glucocorticoids achieve rapid symptom control in most patients yet relapse rates differ markedly once doses fall below 10 mg/day, while responses to azacitidine, JAK-STAT inhibitors or IL-1 blockade are strongly influenced by the presence of concurrent MDS, specific UBA1 variants and the predominance of cutaneous versus cartilaginous disease (60).