EHA 2025 | Using luspatercept in patients with β-thalassemia who convert to transfusion dependence late in life

In this poster, we wanted to highlight our best, our most exciting real-world data from luspatercept, mainly on NTDT patients. So the first message is that we included patients with cardiac comorbidities, such as heart failure or pulmonary hypertension. These patients are usually excluded from clinical trials but of course in everyday practice patients have multiple problems and it seems that they tolerated the treatment well and also achieved sustainable responses. 

Then the second message is that most of these NTDT patients have been diagnosed quite late in life. And this also highlights the fact that this diagnosis is sometimes missed. So we presented 15 patients. Eight of them had started regular transfusions at an advanced age in adulthood. And another seven were about to start regular transfusions because they had several complications. Of course, this patient profile is completely different from a typical TDT patient being transfused from the very first months of their lives, but still, it’s very difficult to introduce a regular transfusion regimen in adult patients. It’s difficult for them to accept it. It’s also quite a burden for healthcare systems. And it’s interesting that these kind of patients with a relatively mild form of thalassemia intermedia or thalassemia NTDT, but also with multiple comorbidities, all responded to the treatment with luspatercept, and they either stopped regular transfusion programs that they had initiated or completely avoided the introduction of a new regular transfusion program, and they all had better hemoglobin levels post-treatment. 

And then among these patients, there were three with alloimmunization, and one of them had the history of a delayed hemolytic transfusion reaction with hyperhemolysis syndrome, and this is a very difficult clinical situation. It is more common in sickle cell disease, but sometimes we do see it in thalassemic patients who are exposed to transfusions quite late as adults, and it’s very difficult to manage. So one of our patients had multiple such episodes, had tried several treatments, and finally responded to luspatercept and has sustained this response for several years now and is off transfusions. Apart from not being transfused in the acute phase of hyperhemolysis, she has maintained her hemoglobin for quite a long time, her hemoglobin response. So this is another message that maybe in this difficult situation of hyperhemolysis, luspatercept can be used and possibly help with the anemia.

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