Beta-thalassaemia causes defective haemoglobin synthesis leading to ineffective
erythropoiesis, chronic haemolytic anaemia, and subsequent clinical complications. Blood …

Purpose β-Thalassemia is an inherited blood disorder characterized by reduced or no
production of adult hemoglobin. Systematic identification of the burden of β-thalassemia with …

Background Patients with transfusion-dependent β-thalassemia need regular red-cell
transfusions. Luspatercept, a recombinant fusion protein that binds to select transforming …

Thalassemia major and sickle cell disease are the two most widely disseminated hereditary
hemoglobinopathies in the world. The outlook for affected individuals has improved in recent …

Objective To estimate a value set for the calculation of Italian-specific quality-adjusted life
years (QALYs), based on preferences elicited on EuroQol five-dimensional (EQ-5D) …

Background Thalassemia is a common disorder worldwide with a predominant incidence in
Mediterranean countries, North Africa, the Middle East, India, Central Asia, and Southeast …

Background Patients with transfusion-dependent thalassemia (TDT) require lifelong blood
transfusions and iron chelation therapy. Thus, patients afflicted with TDT often have to …

Background: Many studies have been conducted in Italy to assess Health-Related Quality-of-
Life (HRQoL) in subjects with different diseases. However, no recent HRQoL population …

Almost 30 years have passed since the first successful hematopoietic stem cell
transplantation in thalassemia and that first patient is now a healthy young adult with a …

Sustained donor engraftment after allogeneic hematopoietic cell transplantation (HCT)
converts to healthy donor hemoglobin synthesis and halts disease symptoms in patients with …