The emerging role of fetal hemoglobin induction in non-transfusion-dependent thalassemia [0.03%]
非输血依赖性地中海贫血的胎儿血红蛋白诱导疗法的新角色
Swee Lay Thein
Swee Lay Thein
Patients with beta (β)-thalassemia who have high levels of fetal hemoglobin (HbF) have less severe anemia and are often transfusion-independent. Therefore, augmentation of HbF production has been a longstanding therapeutic objective. Three...
Elliott Vichinsky
Elliott Vichinsky
Alpha (α)-thalassemia represents a group of recessively inherited hemoglobin disorders marked by deficient or absent synthesis of 1 to all 4 of the α-globin genes. Inactivation of 3 α-globin genes--either by deletional or nondeletional m...
The definition and epidemiology of non-transfusion-dependent thalassemia [0.03%]
非输血依赖性地中海贫血的定义和流行病学
David J Weatherall
David J Weatherall
Inherited hemoglobin-related disorders, which include the structural variants (hemoglobin S, C, and E) and the alpha (α)- and beta (β)-thalassemias, affect more than 300,000 children annually, particularly in malaria-endemic regions stret...
Nancy F Olivieri
Nancy F Olivieri
Hemoglobin E beta (ß)-thalassemia (HbE thalassemia) is a very common form of β-thalassemia that exhibits a heterogeneous clinical presentation and variable clinical course. The reasons for this extraordinary clinical heterogeneity are not...
Ali T Taher,Khaled M Musallam,Mehran Karimi et al.
Ali T Taher et al.
Beta-thalassemia intermedia (TI) is associated with a variety of serious clinical complications that require proactive and comprehensive management. These include skeletal deformities and osteopenia, compensatory extramedullary hematopoiesi...
Maria D Cappellini,Khaled M Musallam,Erika Poggiali et al.
Maria D Cappellini et al.
Beta (β)-thalassemia is characterized by a hypercoagulable state and an increased risk of thrombosis, which can result in significant morbidity and mortality. The molecular and cellular mechanisms contributing to hypercoagulability are div...
Iron overload in non-transfusion-dependent thalassemia: a clinical perspective [0.03%]
非输血依赖性地中海贫血患者铁过载的临床概述
Khaled M Musallam,Maria D Cappellini,John C Wood et al.
Khaled M Musallam et al.
Iron overload due to increased intestinal iron absorption represents an important clinical problem in patients with non-transfusion-dependent thalassemia (NTDT), particularly as they advance in age. Current models for iron metabolism in pat...
The role of ineffective erythropoiesis in non-transfusion-dependent thalassemia [0.03%]
无效红细胞生成在非输血依赖性地中海贫血中的作用
Stefano Rivella
Stefano Rivella
Ineffective erythropoiesis is the hallmark of beta-thalassemia that triggers a cascade of compensatory mechanisms resulting in clinical sequelae such as erythroid marrow expansion, extramedullary hematopoiesis, splenomegaly, and increased g...
Recent advances and treatment challenges in patients with non-transfusion-dependent thalassemia [0.03%]
非输血依赖性地中海贫血患者的治疗进展和挑战
Ali T Taher,Maria D Cappellini,Khaled M Musallam
Ali T Taher
Jecko Thachil
Jecko Thachil
There has been immense progress in the management of venous thromboembolism in recent years with increased awareness and adequate thromboprophylaxis proving successful in reducing the morbidity and mortality associated with this condition. ...