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.