Long-Term Data on Oral Hypomethylating Agents
Explore the latest advancements in oral hypomethylating agents for MDS and CMML, emphasizing their efficacy, patient convenience, and treatment strategies.
In this opening scientific segment, the discussion focuses on recent long-term clinical data evaluating oral hypomethylating agents (HMAs) for myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). Dr. Zeidan reviews new publications and conference presentations reporting extended follow-up from both oral and intravenous HMA therapies. A central focus is the ASCERTAIN trial, which established pharmacokinetic equivalence between oral decitabine/cedazuridine and intravenous decitabine, enabling the oral fixed-dose combination to be approved in the United States.
Dr. Fazal highlights the rationale for using HMAs as first-line therapy in high-risk patients, noting that their mechanism targets disease progression and leukemic transformation. He emphasizes that early and continuous therapy is essential to prevent acquisition of adverse mutations and the development of more aggressive disease phenotypes.
The discussion addresses practical considerations in selecting and administering HMAs. Dr. Fazal notes that oral options, including oral decitabine, provide effective disease control and symptomatic relief, while maintaining flexibility for patients in both community and academic settings. The segment also touches on the integration of supportive care, including management of cytopenias, fatigue, and splenomegaly, as part of holistic treatment planning.
Speakers underscore the importance of planning for potential allogeneic stem cell transplantation in high-risk patients, including HLA typing and early coordination with transplant teams. This proactive approach ensures that patients who progress despite therapy can access advanced interventions promptly.
Finally, the segment highlights emerging strategies for combining oral HMAs with other agents, which may enhance efficacy and expand treatment options. The availability of oral formulations is presented as a pivotal advancement, offering patients greater autonomy, convenience, and the ability to receive care closer to home, while maintaining clinical efficacy equivalent to intravenous therapy.
