Oral Hypomethylating Agents: Adherence, Real-World Data, and Expanding Use

This segment focuses on the practical considerations and evolving use of oral hypomethylating agents (HMAs) in myeloid malignancies, emphasizing adherence, real-world outcomes, and emerging combination therapies. Salman Fazal, MD, addresses concerns about patient adherence with oral therapy vs intravenous administration, noting that continuous treatment is critical in high-risk myelodysplastic syndromes (MDS). Real-world data indicate that interruptions or premature discontinuation of HMAs is common, with up to 44% of patients unable to continue therapy consistently. Oral formulations, particularly those with a condensed 5-day schedule, can improve convenience while maintaining adherence and efficacy comparable to intravenous therapy.

Susan Granato, CRNP, highlights the importance of proactive patient education and support. She describes structured postprescription visits, inclusion of caregivers, and detailed instruction on dosing, adverse effects, and laboratory monitoring, emphasizing the collaborative role of nursing and medical teams in ensuring adherence.

Aditi Shastri, MBBS, discusses the expanding landscape of oral HMAs in combination with agents such as venetoclax for high-risk MDS and acute myeloid leukemia. She describes that oral formulations allow patients to receive effective treatment in community settings, reducing the need for travel to large academic centers while maintaining coordination of care. Preliminary combination data show promising efficacy, suggesting broader adoption and potential “total oral therapy” regimens in the near future.

Overall, this segment highlights the balance between patient convenience, adherence, and clinical outcomes, framing oral HMAs as a practical and effective advancement in modern myeloid malignancy management.