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 …

[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 …

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 …

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 …

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 …

Antibodies against the erythroferrone N-terminal domain prevent hepcidin suppression and ameliorate murine thalassemia

J Arezes, N Foy, K McHugh, D Quinkert… - Blood, The Journal …, 2020 - ashpublications.org
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 …

How I treat non-transfusion-dependent β-thalassemia

AN Saliba, KM Musallam, AT Taher - Blood, 2023 - ashpublications.org
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 …

[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 …

Treating iron overload in patients with non‐transfusion‐dependent thalassemia

AT Taher, V Viprakasit, KM Musallam… - American journal of …, 2013 - Wiley Online Library
Despite receiving no or only occasional blood transfusions, patients with non‐transfusion‐
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 …

KM Musallam, AT Taher, A Kattamis… - Drug Design …, 2023 - Taylor & Francis
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 …