WebJan 12, 2024 · Luspatercept has also been approved recently by the FDA for the treatment of anemia that fails to respond to an erythropoiesis-stimulating agent and requires ≥2 RBC … WebJun 9, 2024 · Thalassaemia is a diverse group of genetic disorders with a worldwide distribution affecting globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin chain production, leading to ineffective erythropoiesis, increased haemolysis, and deranged iron homoeostasis.
Gene therapy for β-thalassaemia: the continuing challenge
WebOct 1, 2010 · Gene therapy for β-thalassaemia: the continuing challenge The β-thalassaemias are inherited anaemias that form the most common class of monogenic disorders in the world. Treatment options are limited, with allogeneic haematopoietic stem cell transplantation offering the only hope for lifelong cure. WebManagement of cardiac complications in thalassemia major hinges on the treatment of the underlying pathophysiology, which often is unmitigated iron overload. The prevalence and predictors of cardiac complications in ‘ex-thalassaemics’ [thalassaemic patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) is unknown at ... cigna hedis
Β-Thalassemias NEJM
WebAn increasing understanding of the underlying molecular and cellular disease mechanisms plus an awareness of limitations of current management strategies are driving research into novel therapeutic options. Here we provide an overview of the current pathophysiology, clinical manifestations, and global burden of beta-thalassaemia. WebThe most anemic patients require regular red blood cell transfusions to avoid death from cardiac failure. However, the inevitable iron accumulation leads to dysfunction, primarily … WebIn addition, the compound heterozygosity of an HPFH deletion with a β 0-thalassaemia mutation can lead to an asymptomatic condition involving mild anaemia ; this is in contrast to the effect of compound heterozygosity of δβ-thalassaemia along with another form of β-thalassaemia that leads to a severe clinical picture and a transfusion ... cigna hepatitis c prior authorization