The 66th Congress of the American Society of Hematology (ASH) highlighted several therapeutic advances in myeloid malignancies. In lower-risk myelodysplastic syndromes (MDS), luspatercept has emerged as an effective alternative to erythropoiesis-stimulating agents, significantly improving transfusion independence. Imetelstat, a recently approved telomerase inhibitor, has demonstrated sustained efficacy in patients refractory to standard treatments. Immunotherapy is advancing with vibecotamab, a bispecific antibody targeting CD123, which has shown promising results in aggressive myeloid neoplasms. The combination of a FLT3 inhibitor, a hypomethylating agent, and venetoclax improves outcomes in FLT3-mutated acute myeloid leukemia (AML). Additionally, menin inhibitors, notably revumenib, offer new therapeutic opportunities for AML with KMT2A rearrangements. Oral formulations, such as ASTX030, facilitate the management of AML and high-risk MDSs. Finally, a novel preclinical model for therapy-related myeloid neoplasms is providing new insights into potential therapeutic targets. These advances mark a significant shift toward more targeted and better tolerated treatments, ultimately improving patient survival and quality of life.
Keywords
Myeloid malignancies, luspatercept, imetelstat, immunotherapy, FLT3, menin inhibitors, oral formulations