When Is the Best Time to Start Erythropoietin-Stimulating Agents for MDS?

Being diagnosed with myelodysplastic syndrome (MDS) poses significant challenges for patients, one of the most common among them is anemia. Erythropoiesis-stimulating agents (ESA) are a standard first-line treatment, but the ideal timing to start this therapy remains unclear. 

Recent updates of the EPO-PRETAR clinical trial provided new insights into how the timing of ESA treatment impacts outcomes for lower-risk MDS patients. In this article, you’ll learn the highlights and results presented. 

When is Best to Initiate Erythropoiesis-Stimulating Agents? 

Should ESA be initiated early, before patients become dependent on red blood cell (RBC) transfusions, or later when transfusion dependence has developed?

To explore this question, the trial enrolled 84 patients with lower-risk MDS and anemia, and divided them into two groups:

1. Early Onset: ESA treatment began within six months of diagnosis.
2. Late Onset: ESA was only given when hemoglobin levels dropped below a predefined threshold for transfusion.

Patients in both groups received weekly doses of 60,000 IU of erythropoietin Alfa for at least 12 weeks. Key inclusion criteria included baseline hemoglobin (Hgb) levels between 9 and 10.5 g/dL. 

Patients were followed for a median of 34 months to evaluate outcomes such as time to red blood cell transfusion dependence, progression to higher-risk MDS or acute myeloid leukemia (AML), and overall survival.

Key Findings

• Improvement in blood cell production:
  • 79.5% of patients in the early-onset group achieved hematologic improvement, compared to 54% in the late-onset group.
• Disease Progression and Survival:
  • The progression rates to higher-risk MDS or AML were similar in both groups.
  • Median overall survival was 49.6 months in the early group and 55 months in the late group.
• Lab Values as Predictors of Response:
  • Patients with lower serum erythropoietin levels (<50 IU/L) were more likely to improve blood cell production in the bone marrow.
  • Patients with specific genetic mutations, such as SF3B1, had better responses to ESA.

What Does This Mean for the Future of MDS Supportive Treatment?

Findings from the EPO-PRETAR trial suggest that initiating erythropoietin-stimulating agents early in lower-risk MDS patients with anemia can lead to longer-lasting improvements in blood counts. 

Importantly, lab findings such as serum erythropoietin levels, genetic mutations, and immune-related gene expression patterns may help predict which patients are most likely to benefit from ESA therapy.

These results highlight the importance of discussing treatment timing and options with your healthcare team. Early treatment could provide a longer period of relief from anemia-related symptoms.

Why Participating in Research Matters

This study shows the critical role of clinical trials in advancing our understanding of MDS and improving patient care. By participating in research, people with MDS contribute to discoveries that may shape future treatment strategies and offer hope for more personalized approaches to managing the cancer. 

If you or a loved one has MDS, consider asking your healthcare team about opportunities to participate in clinical trials or learn more by using HealthTree’s clinical trial finder, a great free resource for browsing and filtering to suit your preferences. Together, we can work toward better outcomes for all MDS patients.

Keep learning about more clinical trial updates and research advances written just for you! 

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Source: 

• Early Versus Late Onset of ESA in Lower Risk Anemic MDS Patients: Results of the GFM Randomized Phase III EPO-Pretar Trial