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ASH 2025 | The real-world experience with luspatercept for thalassemia treatment
In this video, Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, discusses the real-world experience with luspatercept in patients with thalassemia, noting that outcomes are comparable to those observed in clinical trials. Prof. Cappellini also highlights that fetal hemoglobin (HbF) may serve as a biomarker of response, with patients who have higher HbF levels responding better to therapy. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
Luspatercept has been already approved in many countries and so now it is used in real life. So I think I can comment on the recent data published on real life. These are collected data in different centers. We have a huge study conducted in Italy including more than 10 centers and the outcomes are very similar to those observed in the clinical trial. So approximately the responders for the primary endpoint, which is for the transfusion, for BELIEVE it is a reduction of equal or more than 33% transfusion burden compared to baseline...
Luspatercept has been already approved in many countries and so now it is used in real life. So I think I can comment on the recent data published on real life. These are collected data in different centers. We have a huge study conducted in Italy including more than 10 centers and the outcomes are very similar to those observed in the clinical trial. So approximately the responders for the primary endpoint, which is for the transfusion, for BELIEVE it is a reduction of equal or more than 33% transfusion burden compared to baseline. For BEYOND, it’s the increase of hemoglobin of one gram or 1.5 compared to baseline. So even in real life, we achieve similar results. Unfortunately, we don’t have yet sufficient data for NTDT because it has been approved in Europe for NTDT, not yet by the FDA, and there are no available real-life data. But for BELIEVE, for the transfusion-dependent patients, we have a lot of data supporting a good response in almost 30-40% of the patients. And looking more carefully at what could be some biomarkers of response, from this real-life paper, it comes out that fetal hemoglobin may be a marker of response. Those patients who start with a higher level of fetal hemoglobin are responding better. So what we are doing these days is trying to pull together the data to do a multivariate analysis in order to identify a profile of patients who could be a good candidate to respond.
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