Thalassaemia
AT Taher, DJ Weatherall, MD Cappellini - The Lancet, 2018 - thelancet.com
Inherited haemoglobin disorders, including thalassaemia and sickle-cell disease, are the
most common monogenic diseases worldwide. Several clinical forms of α-thalassaemia and …
most common monogenic diseases worldwide. Several clinical forms of α-thalassaemia and …
[HTML][HTML] Non-transfusion-dependent thalassemias
KM Musallam, S Rivella, E Vichinsky… - …, 2013 - ncbi.nlm.nih.gov
Non-transfusion-dependent thalassemias include a variety of phenotypes that, unlike
patients with beta (β)-thalassemia major, do not require regular transfusion therapy for …
patients with beta (β)-thalassemia major, do not require regular transfusion therapy for …
How I manage medical complications of β-thalassemia in adults
AT Taher, MD Cappellini - Blood, the Journal of the American …, 2018 - ashpublications.org
The complex pathophysiology in β-thalassemia can translate to multiple morbidities that
affect every organ system. Improved survival due to advances in management means that …
affect every organ system. Improved survival due to advances in management means that …
Management of iron overload in beta-thalassemia patients: clinical practice update based on case series
VM Pinto, GL Forni - International Journal of Molecular Sciences, 2020 - mdpi.com
Thalassemia syndromes are characterized by the inability to produce normal hemoglobin.
Ineffective erythropoiesis and red cell transfusions are sources of excess iron that the human …
Ineffective erythropoiesis and red cell transfusions are sources of excess iron that the human …
Prevalence and risk factors for pulmonary arterial hypertension in a large group of β-thalassemia patients using right heart catheterization: a Webthal study
G Derchi, R Galanello, P Bina, MD Cappellini, A Piga… - Circulation, 2014 - Am Heart Assoc
Background—Pulmonary arterial hypertension (PAH) remains a concern in patients with β-
thalassemia major (TM) and intermedia (TI); however, studies evaluating its prevalence and …
thalassemia major (TM) and intermedia (TI); however, studies evaluating its prevalence and …
Antibodies against the erythroferrone N-terminal domain prevent hepcidin suppression and ameliorate murine thalassemia
Erythroferrone (ERFE) is produced by erythroblasts in response to erythropoietin (EPO) and
acts in the liver to prevent hepcidin stimulation by BMP6. Hepcidin suppression allows for …
acts in the liver to prevent hepcidin stimulation by BMP6. Hepcidin suppression allows for …
How I treat 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 …
ineffective erythropoiesis in non-transfusion-dependent β-thalassemia (NTDT) results in a …
[HTML][HTML] Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia
KM Musallam, A Vitrano, A Meloni, SA Pollina… - …, 2021 - ncbi.nlm.nih.gov
HCC: hepatocellular carcinoma. α/β-globin imbalance leading to clinically-significant
disease). For each patient, data were retrieved for gender, age at last observation, status …
disease). For each patient, data were retrieved for gender, age at last observation, status …
Treating iron overload in patients with non‐transfusion‐dependent thalassemia
Despite receiving no or only occasional blood transfusions, patients with non‐transfusion‐
dependent thalassemia (NTDT) have increased intestinal iron absorption and can …
dependent thalassemia (NTDT) have increased intestinal iron absorption and can …
Profile of Luspatercept in the Treatment of Anemia in Adults with Non-Transfusion-Dependent β-Thalassemia (NTDT): Design, Development and Potential Place in …
Over the past decade, evidence has been mounting on the detrimental clinical sequelae of
untreated anemia in patients with non-transfusion-dependent β-thalassemia (NTDT). There …
untreated anemia in patients with non-transfusion-dependent β-thalassemia (NTDT). There …