Underutilization of Effective Treatments in High-Risk Myelodysplastic Syndromes Revealed by New Study

A recent comprehensive analysis highlights a concerning trend in the treatment of high-risk myelodysplastic syndromes (MDS), a group of blood cancers that predominantly affect older adults. Despite the availability of hypomethylating agents (HMAs) — such as azacitidine and decitabine — which have been shown in clinical trials to extend survival and improve quality of life, many patients are not receiving these guideline-recommended therapies.

The study, the largest of its kind in the United States, examined Medicare claims data from over 49,000 older adults diagnosed with MDS. Findings indicate that only about 16% of newly diagnosed high-risk MDS patients received HMAs during the analysis period (2011–2014). This figure is significantly lower than the estimated 30–40% of high-risk patients who should be prescribed these drugs. Furthermore, even among patients who initiated HMA treatment, many failed to complete the recommended course of therapy, with over one-third discontinuing after four cycles and half by six cycles.

The research also uncovered disparities in treatment access. Women and nonwhite patients were markedly less likely to receive HMAs compared to their white and male counterparts. Specifically, women were 19% less likely, and Black patients 30% less likely, to start HMA therapy. Older patients over 85 and those living in underserved areas also had lower treatment rates.

Dr. Sudipto Mukherjee, the study’s lead author, emphasized the importance of delivering full, guideline-adherent treatment regimens. He explained that the common practice of prematurely stopping therapy, often in response to initial blood count declines or fatigue, hampers the potential benefits of HMAs. Proper management and persistence through treatment cycles are crucial for optimal outcomes.

The study underlines the need for improved strategies to address these treatment gaps. Potential solutions include partnerships between community clinics and specialized centers to facilitate expert guidance on HMA use, dosage, and management of side effects. While the study was based on retrospective Medicare data, limiting insights into the exact reasons for treatment discontinuation, its findings underscore an urgent need to enhance therapy delivery for high-risk MDS patients.

Overall, the research reveals that despite the proven benefits of hypomethylating agents, a significant number of patients do not receive or complete optimal treatment, which may contribute to the stagnant survival rates seen over the past two decades. Addressing these disparities and treatment adherence issues is essential to improving patient outcomes in high-risk MDS.

Source: https://medicalxpress.com/news/2025-08-patients-high-myelodysplastic-syndromes-effective.html