ASH 2024 | The EPO-Pretar trial: early versus late onset of ESA in LR-MDS

I will talk about two abstracts from the Groupe Francophone des Myelodysplasies. So the first one is the EPO-Pretar trial, where the background is that ESA has authorization for MDS patients with anemia in Europe. But the time for introduction of ESA early or late onset is not really known. And in a retrospective study, we have shown that the time to red blood cell transfusion dependence is longer in patients who had early onset of ESA within six months from diagnosis. So in the trial we presented at ASH this year, we evaluated prospectively the early or late onset of ESA on the time to red blood cell transfusion dependence. So therefore we included the patients, lower risk MDS patients with hemoglobin between 9 to 10.5 grams per deciliter in the early arm. And in the late arm, the patients had ESA whenever hemoglobin reached 8 to 9 grams per deciliter. So there were 43 patients in the early arm, 41 patients in the late arm, and they were well balanced in terms of gender, IPSS-R score and WHO classifications. And the primary endpoint was the time to red blood cell transfusion dependence and it was the same between both arms with a median time to red blood cell transfusions of 36 months. Nevertheless, for the secondary endpoint we evaluated the erythroid response and duration of response to ESA and the erythroid response was higher in the early arm with 79% of response rate in the early arm versus 54% in the late arm. And the duration of response was longer in the early arm, 30 months versus 12 months in the late arm. These results did not translate into quality of life scores. The quality of life was the same in both arms and for longer outcome, PFS and overall survival, they were the same in both arms. In biological correlative studies, we have evaluated the IPSS-M score and in the responders, they had a lower IPSS-M score. And in terms of red blood cell transfusion dependence time, it was shorter in the patients with a high IPSS-M score. And finally, we have done RNA-Seq and cytokine profile dosing and we have shown interestingly by RNA-Seq that there was a hereditary signature which predicted the patients who will become red blood cell transfusion dependent and the responders to ESA had a lower IL-6 cytokine dosage. So finally, this abstract demonstrates at the data cutoff of July 2024 where half of the patients had ESA in the late arm that the red blood cell transfusion dependence was the same in early or late onset arm of ESA with the same outcomes, the same quality of life and with interesting biological markers predicting time to transfusion dependence.

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