The Long-Term Efficacy of Erythropoiesis-Stimulating Agents in Patients With Low-Risk or Intermediate-1-Risk Myelodysplastic Syndrome: Multicenter Real-Life Data.

OBJECTIVE: To evaluate the long-term clinical efficacy of epoetin alfa and darbepoetin alfa in patients with myelodysplastic syndromes (MDS) in the real-life setting.

MATERIALS AND METHODS: A total of 204 patients with low-risk or intermediate-1-risk MDS who received epoetin alfa or darbepoetin alfa were included. Hemoglobin levels and transfusion need were recorded before and during 12-month, 24-month, 36-month and 48-month treatment.

RESULTS: At 36-month (p=0.025) and 48-month (p=0.022) visits, epoetin alfa vs. darbepoetin alfa yielded significantly higher hemoglobin levels. Transfusion-need was also significantly lower in epoetin alfa vs. darbepoetin alfa groups at 24-month (p=0.012), and in low risk vs. intermediate risk groups at 24-month (p=0.018), 36-month (p=0.025) and 48-month (p<0.001) visits. Treatment response rates at 24-month, 36-month and 48-month visits in epoetin alfa (43.0, 33.6 and 27.1%), darbepoetin alfa (29.9, 22.7 and 16.5%), low risk (39.3, 30.0 and 26.0 %) and intermediate risk (29.6, 24.1 and 11.1%) groups were lower than 12-month response rates, significantly at 36-month and 48-month visits (p ranged <0.05 to <0.001).

CONCLUSION: This real-life long-term ESA extension study investigated the clinical efficacy of epoetin alfa and darbepoetin alfa for up to 48 months, revealed the treatment efficacy to reach plateau starting from the 24th month of therapy with a continuing decrease in treatment response rates, regardless of treatment type, risk status or gender. Nonetheless, significantly higher hemoglobin levels and marked improvement in transfusion-need was evident in the epoetin-treated vs. darbepoetin-treated groups and in the low risk vs. intermediate risk groups.