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ASH 2022 | MEDALIST: episodes of transfusion independence in patients with LR-MDS treated with luspatercept

Uwe Platzbecker, MD, University of Leipzig, Leipzig, Germany, discusses results from the Phase III MEDALIST trial (NCT02631070), which investigated the use of luspatercept for patients with low-risk myelodysplastic syndromes (LR-MDS) who are transfusion dependent. The primary endpoint of transfusion independence (TI) was met, and it was shown that patients who experience TI after a period of time and receive a subsequent transfusion should continue luspatercept therapy to increase the chance of several consecutive episodes of TI. This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

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Transcript (edited for clarity)

This abstract is about the MEDALIST trial. The MEDALIST trial was a randomized placebo-controlled study, investigating luspatercept in transfusion-dependent patients with a ring sideroblastic phenotype. The study at the end led to the approval of luspatercept in ring sideroblastic patients failing ESA therapy, or not being eligible to ESA therapy because of endogenous EPO level.

The primary endpoint was met, with regards to transfusion independence...

This abstract is about the MEDALIST trial. The MEDALIST trial was a randomized placebo-controlled study, investigating luspatercept in transfusion-dependent patients with a ring sideroblastic phenotype. The study at the end led to the approval of luspatercept in ring sideroblastic patients failing ESA therapy, or not being eligible to ESA therapy because of endogenous EPO level.

The primary endpoint was met, with regards to transfusion independence. This poster, now at this ASH meeting, is about the multiple episodes of transfusion independence. What does it mean? It means that patients on luspatercept receive transfusion independence. And at the end of the day, after a couple of weeks or sometimes many months, another transfusion event occurred. However, treatment was continued in order to show whether a second, or sometimes third, episode of transfusion independence occurred. And this was the case, and is actually the main content of this poster.

So if you have a patient with luspatercept with a ring sideroblastic phenotype, and this patient experiences a transfusion independence after a certain period of time, and then a consecutive transfusion event; it is worth continuing the therapy in order to observe a consecutive second or third, sometimes even fourth, period of transfusion independence in these patients.

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Disclosures

Geron: Honoraria; Jazz: Honoraria; Abbvie: Honoraria; BMS/Celgene: Honoraria; Takeda: Honoraria; Novartis: Honoraria; Silence Therapeutics: Honoraria; Janssen: Honoraria.