Pevonedistat

Despite challenges in improving treatment for high-risk myelodysplastic syndrome (HR-MDS), advancements in combination therapies and molecular profiling offer hope. Allogeneic transplantation remains the only curative option, but posttransplant relapse is a concern. Recent trials combining azacitidine with various drugs showed promise but failed to outperform monotherapy in phase 3. Future potential lies in targeted treatments like azacitidine with venetoclax or tamibarotene, and refining response criteria. Molecular findings are crucial for more precise treatment design.