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] β-Thalassemia
R Origa - Genetics in Medicine, 2017 - Elsevier
Abstract β-Thalassemia is caused by reduced (β+) or absent (β 0) synthesis of the β-globin
chains of hemoglobin. Three clinical and hematological conditions of increasing severity are …
chains of hemoglobin. Three clinical and hematological conditions of increasing severity are …
β-Thalassemias
AT Taher, KM Musallam… - New England Journal of …, 2021 - Mass Medical Soc
β-Thalassemias Defective synthesis of the β-globin chain causes recessively inherited
disorders characterized by inadequate hemoglobin production and chronic anemia …
disorders characterized by inadequate hemoglobin production and chronic anemia …
Serum ferritin is an important inflammatory disease marker, as it is mainly a leakage product from damaged cells
DB Kell, E Pretorius - Metallomics, 2014 - academic.oup.com
Abstract" Serum ferritin" presents a paradox, as the iron storage protein ferritin is not
synthesised in serum yet is to be found there. Serum ferritin is also a well known …
synthesised in serum yet is to be found there. Serum ferritin is also a well known …
[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 …
2021 update on clinical trials in β‐thalassemia
KM Musallam, R Bou‐Fakhredin… - American journal of …, 2021 - Wiley Online Library
The treatment landscape for patients with β‐thalassemia is witnessing a swift evolution, yet
several unmet needs continue to persist. Patients with transfusion‐dependent β …
several unmet needs continue to persist. Patients with transfusion‐dependent β …
Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo …
AT Taher, MD Cappellini, A Kattamis… - The Lancet …, 2022 - thelancet.com
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 …
concentrations lower than 10 g/dL are associated with a higher risk of morbidity, mortality …
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 …
Guidelines for quantifying iron overload
JC Wood - Hematology 2014, the American Society of …, 2014 - ashpublications.org
Both primary and secondary iron overload are increasingly prevalent in the United States
because of immigration from the Far East, increasing transfusion therapy in sickle cell …
because of immigration from the Far East, increasing transfusion therapy in sickle cell …
Estimating tissue iron burden: current status and future prospects
JC Wood - British journal of haematology, 2015 - Wiley Online Library
Iron overload is becoming an increasing problem as haemoglobinopathy patients gain
greater access to good medical care and as therapies for myelodysplastic syndromes …
greater access to good medical care and as therapies for myelodysplastic syndromes …