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General Updates | Long-term follow-up of the BEYOND trial: luspatercept in non-transfusion dependent thalassemia
Ali Taher, MD, PhD, FRCP, American University of Beirut Medical Center, Beirut, Lebanon, presents the long-term results of the Phase II BEYOND study (NCT03342404), which evaluated the efficacy and safety of luspatercept in patients with non-transfusion dependent beta-thalassemia (NTDT). Prof. Taher highlights that the study showed a sustained increase in hemoglobin levels in patients treated with luspatercept, with around 95% achieving an increase of more than one gram per deciliter, and a consistent safety profile with no new safety findings. This interview took place virtually.
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Transcript
As you all know, chronic anemia due to non-transfusion dependent beta-thalassemia in the NTDT patient, the patients that were included in the BEYOND trial, result in a lot of clinical morbidities, particularly with inadequate management. As you all know, luspatercept was previously shown to improve hemoglobin levels in patients with NTDT in the Phase II randomized double-blind placebo-controlled BEYOND trial that was published previously...
As you all know, chronic anemia due to non-transfusion dependent beta-thalassemia in the NTDT patient, the patients that were included in the BEYOND trial, result in a lot of clinical morbidities, particularly with inadequate management. As you all know, luspatercept was previously shown to improve hemoglobin levels in patients with NTDT in the Phase II randomized double-blind placebo-controlled BEYOND trial that was published previously. This study now reports only the long-term efficacy and safety results from the final analysis of the previously discussed BEYOND trial, which spans around 2.2 years of follow-up.
The median treatment duration was, as I said, 2.28 years of luspatercept. The overall, we had around 95% and 22% of patients in the luspatercept and placebo arms, respectively, achieved an increase in hemoglobin level of more than one gram per deciliter. The patient-reported tiredness and weakness showed sustained improvement in the luspatercept treatment arm. Most of the treatment-emergent adverse events that you would like to know about were headache, bone pain, back pain, and arthralgia. An important treatment-emergent issue was the extramedullary hematopoiesis event – we reported this in around 9% in the luspatercept arm versus 2% in the placebo arm. Although the differences in treatment exposure prevented a proper comparative threat. Of the four patients receiving luspatercept who reported thromboembolic events, all had more than one risk to have thrombotic events.
So these results, the long-term safety and efficacy, show that luspatercept led to a sustained increase in hemoglobin level in the NTDT group up to 4.6 years of treatment with a consistent safety profile and no new safety findings. So it is a valuable treatment option for our patients and it addresses the need for effective long-term treatment and management of anemia in our patients.
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