Treatment Disparities and Poor Adherence in High-Risk MDS

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 key findings of a 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.

First, based on current guidelines, all patients with high-risk MDS should receive HMAs. High-risk MDS is found in 30% to 40% of all newly diagnosed patients with MDS. However, the study cohort showed that only 16% of these patients actually received HMAs. This is a very low number, suggesting that almost half of newly diagnosed high-risk MDS patients are not receiving the treatment they are indicated for. This is a troubling finding, as outcomes will be worse if patients are not treated. The finding reinforces similar reports from previous investigators who found treatment rates between 14% and 18%, placing the study right in that same range.

Second, investigators looked at factors influencing who receives treatment. They found that patients most likely to receive HMA were younger, white males, with "younger" defined as being between 65 and 74 years of age. By contrast, patients older than 74, females, and Black patients and those of other ethnicities were significantly less likely to receive HMAs, even with a high-risk MDS diagnosis. The reasons for these disparities are not entirely clear and require a much deeper investigation to find the real answers. This information will help investigators find ways to address these disparities and ensure everyone receives the treatment they need.

Third, the study looked at adherence to the recommended treatment guidelines. To achieve maximum therapeutic benefit, patients on HMAs are supposed to complete at least 4 to 6 treatment cycles. A cycle is a 4-week period, or roughly 28 days. For azacitidine, this means taking the drug for 7 consecutive days, and for decitabine, it's for 5 consecutive days. The cohort showed a wide divergence from these guidelines. Not only were 50% of the patients off treatment by cycle 6, but 50% also did not even receive a complete cycle's worth of doses. The criteria for a complete cycle were at least 4 of the 7 daily doses for azacitidine and at least 3 of the 5 daily doses for decitabine. If patients are not getting the right treatment in the right way and for the right duration, they will not achieve the best possible outcomes.

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