Non-Transfusion Dependent Thalassaemias
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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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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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Scientific 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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Scientific Literature
Survival and Causes of Death in 2,033 Patients with Non-Transfusion-Dependent β-Thalassemia
Non-transfusion-dependent β-thalassemia (NTDT) is a broad term encompassing patients who do not require lifelong transfusion therapy for survival. Our understanding of the disease process in NTDT has evolved significantly over…
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