Scientific Literature
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Association of Sickle Cell Disease With Severe Maternal Morbidity
Objective: To evaluate the association between sickle cell disease (SCD) and severe maternal morbidity (SMM) in a contemporary cohort of deliveries by non-Hispanic Black people. Methods: We retrospectively examined SMM…
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Juggling Between the Cost and Value of New Therapies: Does Science Still Serve Patient Needs?
Thalassaemia International Federation (TIF), representing the united voice of people with thalassaemia and their families globally, has been striving for more than three decades to empower research, by academic communities…
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Overall and Complication‐free Survival in a Large Cohort of Patients With β‐Thalassemia Major Followed Over 50 Years
We report data on survival and complications for a longitudinal cohort of 709 transfusion-dependent β-thalassemia major patients (51.1% males) born between 1970 and 1997 and followed through 2020 at seven major…
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Emergent Treatments for β-Thalassemia and Orphan Drug Legislations
Highlights • β-THAL is one of the most prevalent blood disorders in some regions of the world. • However, given its prevalence, in the EU and the US it is classified as…
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Renal Dysfunction in Pediatric Patients in Iraq With β-Thalassemia Major and Intermedia
Background: With optimum transfusion and chelation therapy, the survival of β-thalassemia patients and the incidence of various complications, including renal complications, have improved. Objectives: To investigate renal involvement in β-thalassemia…
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Precision Medicine and Sickle Cell Disease
Sickle cell disease (SCD) is characterized by variable clinical outcomes, with some patients suffering life-threatening complications during childhood, and others living relatively symptom-free into old age. Because of this variability,…
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Does Hepcidin Tuning Have a Role Among Emerging Treatments for Thalassemia?
The treatments available for thalassemia are rapidly evolving, with major advances made in gene therapy and the modulation of erythropoiesis. The latter includes the therapeutic potential of hepcidin tuning. In…
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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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Nutritional Status in a Sample of Patients With β-Thalassemia Major
Patients suffering from thalassemia have decreased levels of lean body mass and an increased nutritional risk. The aim of this study was to assess the body composition and vitamin D…
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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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