Why HMA Treatment for MDS Fails to Meet Expectations

In an interview with Targeted Oncology, Sudipto Mukherjee, MD, PhD, MPH, Leukemia Program, Department of Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, discusses the implications and future directions of recent study published in Blood Neoplasia investigating disparities in real-world treatment patterns of hypomethylating agents (HMAs) among patients with myelodysplastic syndromes (MDS) in the US.

In the future, studies are needed to understand why these disparities exist and why there's such a wide divergence from recommended treatment guidelines. Mukherjee believes that underlying knowledge gaps and inherent biases are hindering the delivery of effective care. A mixed-methods approach, which includes interviewing both doctors and patients, could help researchers understand the reasons behind delayed treatment, nontreatment, or premature discontinuation of therapy.

It's important to remember that the findings primarily reflect treatment patterns in community settings, not in university hospitals or major academic cancer centers. This is because Medicare data, which was primarily used for the study, largely covers patients diagnosed and treated in community practices. The diagnosis of MDS is complex, and the treatment, which involves periodic cycles of chemotherapy over a long period, can be burdensome for both patients and providers. For a community oncologist who is a generalist and sees a wide variety of cancers, it can be difficult to dedicate a disproportionate amount of time to a single, rare disease like MDS.

One solution, based on evidence from other rare conditions, is for patients with high-risk MDS to get a one-time consultation with an MDS expert from a nearby large hospital or academic center. Once a care plan is established, the local oncologist can follow it, and the patient's care can be shared between the 2 doctors. Researchers believe this would help mitigate many of the barriers we observed and improve outcomes.

Researchers also need to understand why there are age, gender, and racial disparities in treatment. While it's not surprising that certain racial groups, such as White patients, often have better access and outcomes in cancer care, there need to be disease-specific factors that are influencing who gets timely treatment. A mixed-methods approach that combines qualitative and quantitative interviews with patients and physicians at different time points could provide a comprehensive understanding of the issues.

Mukherjee is hoping these findings will lead to new studies to fix these problems. Unfortunately, the field is lagging behind in terms of new therapies for MDS, particularly for newly diagnosed high-risk patients. For 2 decades, there have only been 2 drugs, so clinicians must make the most of them. Even when newer drugs become available, they will likely be built on the backbone of HMAs, reinforcing the need to improve how clinicians use them today.

This description was generated with assistance from Google Gemini. It was edited and reviewed by Targeted Oncology staff. If you have any questions about the use of AI, please contact us.

REFERENCE:

Mukherjee S, Dong W, Gerds AT, et al. Disparities in Real-World Treatment Patterns of Hypomethylating Agents Among Patients with Myelodysplastic Syndromes in the US. Blood Neoplasia 2025; 100156. doi: https://doi.org/10.1016/j.bneo.2025.100156