Researchers have identified age-, sex-, and race-related disparities around treatment with hypomethylating agents for patients with myelodysplastic syndromes in the United States. The receipt of hypomethylating agents was found to favor younger, male, White patients, according to findings published in Blood Neoplasia.
Additionally, the study authors found that the duration of hypomethylating agent therapy varied, with many not completing the recommended number of cycles of treatment in line with clinical guidelines.
“The disparities we found based on gender, race, and ethnicity were really striking,” said the study’s lead author, Sudipto Mukherjee, MD, PhD, MPH, Physician in the Department of Hematology and Medical Oncology at Cleveland Clinic. “Given the absence of newly approved therapies over the past 2 decades, the most impactful way to improve outcomes in newly diagnosed high-risk myelodysplastic syndrome requires that we do better with the available therapies. Making changes with these therapies and how they are given (that is, when to treat and how to treat) is a key intervention that can have a huge impact.”
Study Methods and Rationale
Real-world findings of patients with myelodysplastic syndromes have shown a decreased effectiveness of hypomethylating agents, even though it is the best available treatment option, especially for older patients with high-risk disease who are ineligible for a bone marrow transplant. The researchers analyzed patterns and durations of use to gain a greater understanding of why that is, especially when there is a lack of newer, effective agents for myelodysplastic syndromes.
They conducted a retrospective cohort study of 49,514 patients who were at least 65 years old with incident myelodysplastic syndrome between 2012 and 2013. The eligible individuals were found using 2011–2014 Medicare claims data. This comprised the largest analysis of myelodysplastic syndrome treatment patterns in the United States to date as well as the most comprehensive study of real-world hypomethylating agent usage.
Key Study Findings
Of all evaluated patients with myelodysplastic syndromes, only 16.1% received hypomethylating agents, even though about 30% to 40% of patients would have been high risk at diagnosis, and therefore eligible for treatment with hypomethylating agents.
Adjusted analyses showed that patients 85 years or older had the lowest likelihood of receiving hypomethylating agents (adjusted odds ratio [aOR] = 0.41; 95% confidence interval [CI] = 0.38–0.44). Women had lower odds for receiving hypomethylating agents than men (aOR = 0.81; 95% CI = 0.77–0.86), and Black patients also had lower odds than White patients (aOR = 0.70; 95% CI = 0.62–0.8).
Among patients who did receive hypomethylating agents, they were less likely to receive a full course of at least 4 cycles of therapy if they were treated with decitabine (aOR = 0.7; 95% CI = 0.62–0.78), had two to three cytopenias (aOR = 0.69; 95% CI = 0.61–0.78), were nursing home residents (aOR = 0.64; 95% CI = 0.46–0.90), or had high frailty (aOR = 0.50; 95% CI = 0.34–0.75).
“If you are not even treating the patients for the recommended duration of time, you will not see a response,” said Dr. Mukherjee. “When starting these treatments, blood counts and transfusion needs may initially get worse before they improve, and you have to plow through it. That is not the time to discontinue, but that is what the data are saying.” He suggested that some clinicians or patients may be too quick to stop treatment or skip doses as a result of these early adverse effects.
Dr. Mukherjee recommended that community health clinics partner with larger tertiary care centers to guide hypomethylating agent treatment for these patients to overcome these gaps in care.
Disclosure: For full disclosures of the study authors, visit sciencedirect.com.
