Non-Transfusion Dependent Thalassaemias
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Scientific Literature
Patient-Reported Outcomes With Luspatercept Through 5 Years of Treatment in Patients With Non-Transfusion-Dependent β-Thalassemia Treated in the BEYOND Trial
Abstract In the phase 2, double‐blind, randomized controlled BEYOND trial (NCT03342404), luspatercept increased hemoglobin levels in patients with non‐transfusion‐dependent β‐thalassemia (NTDT). This study assessed long‐term effects of luspatercept on patient‐reported…
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Scientific Literature
Phase 2a Randomized Study to Evaluate Sapablursen in Patients With Non-Transfusion Dependent Β-Thalassemia Intermedia
Non-transfusion-dependent β-thalassemia intermedia (NTDT) is a genetically heterogeneous disorder characterized by decreased β-globin production in erythroid cells resulting from mutations in the β-globin gene that is not treated with transfusion.…
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Scientific Literature
Impact of Lifetime Anaemia and Iron Control on Outcomes in Β-Thalassaemia: Data from the Longitudinal de-LIGHT Study
Summary Evidence on the impact of anaemia and iron control on morbidity and mortality in β‐thalassaemia is limited to short‐term studies assessing spot measures. We conducted a retrospective longitudinal cohort…
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Scientific Literature
‘Phenoconversion’ in Adult Patients with β-Thalassemia
Rate and risk factors for phenoconversion from non-transfusion-dependent β-thalassemia (NTDT) to transfusion-dependent β-thalassemia (TDT) during a 10-year follow up of adult patients in Italy.
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Scientific Literature
How I Τreat Non-Transfusion-Dependent β-Thalassemia
The intricate interplay of anemia and iron overload under the pathophysiological umbrella of ineffective erythropoiesis in non-transfusion-dependent β-thalassemia (NTDT) results in a complex variety of clinical phenotypes that are challenging…
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TIF News
NEW PUBLICATION | Guidelines for the Management of Non-Transfusion-Dependent β-Thalassaemia
This novel edition is an invaluable contribution in upgrading the care of individuals with NTDT, a condition that has been underestimated in importance and clinical significance for many years. TIF…
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Clinical News
TOP STORY | EU Approves Luspatercept for Anemia in Patients with Non–Transfusion-Dependent β-Thalassaemia
The approval was based on findings from the phase 2 BEYOND trial, which demonstrated that 77.1% of patients treated with luspatercept (n = 74/96) experienced a mean haemoglobin (Hb) increase…
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Scientific Literature
Luspatercept for the Treatment of Anaemia in Non-Transfusion-Dependent β-Thalassaemia (BEYOND): A Phase 2, Randomised, Double-blind, Multicentre, Placebo-controlled Trial
Background In patients with non-transfusion-dependent β-thalassaemia, haemoglobin concentrations lower than 10 g/dL are associated with a higher risk of morbidity, mortality, and impaired quality of life. No drugs are specifically…
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Scientific Literature
Safety and Efficacy of Mitapivat, an Oral Pyruvate Kinase Activator, in Adults with Non-Transfusion Dependent α-Thalassaemia or β-Thalassaemia: An Open-label, Multicentre, Phase 2 Study
Background: Patients with non-transfusion-dependent thalassaemia (NTDT), although they do not require regular blood transfusions for survival, can still accrue a heavy burden of comorbidities. No approved disease-modifying therapies exist for these…
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Scientific Literature
Iron Overload Status in Patients with Non-Transfusion-Dependent Thalassemia in China
Background: Non-transfusion-dependent thalassemia (NTDT) is a genetic disorder most commonly including beta-thalassemia intermedia (Beta-TI), HbE/Beta thalassemia (HbE/Beta thalassemia), and hemoglobin H disease (HbH disease). NTDT patients can be at risk of…
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