Catherine Skari

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…

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BREAKING NEWS | Bluebird’s Gene Therapy For β-Thalassaemia Wins US Approval

The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s Chief Commercial Officer, said in…

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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…

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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…

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New Entity—Thalassemic Endocrine Disease: Major Beta-Thalassemia and Endocrine Involvement

Beta-thalassemia (BTH), a recessively inherited haemoglobin (Hb) disorder, causes iron overload (IO), extra-medullary haematopoiesis and bone marrow expansion with major…

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2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia

Beta-thalassemia and particularly its transfusion-dependent form (TDT) is a demanding clinical condition, requiring life-long care and follow-up, ideally in specialized…

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A Short Guide for the Management of Transfusion-Dependent Thalassaemia (2nd edition – 2022)

Prepared by: Farmakis, D. Reviewed by: Eleſtheriou, A., Bain, B.

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Frequency, Pattern, and Associations of Renal Iron Accumulation in Sickle/β-Thalassemia Patients

We evaluated frequency, pattern, and associations of renal iron accumulation in sickle/β-thalassemia. Thirty-three sickle/β-thalassemia patients (36.5 ± 14.7 years; 13 females), 14 homozygous…

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TPFS Jointly Celebrates ITD2022 and Silver Jubilee Anniversary

Under the slogan ”Hope Expands”, the Society planned multiple offline and online events aimed at reflecting the progress achieved in…

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Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective

A significant amount of attention has recently been devoted to the mechanisms involved in hemoglobin (Hb) switching, as it has…

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