Patients with non-transfusion-dependent thalassemia (NTDT) experience many clinical complications despite their independence from frequent transfusions. Morbidities in NTDT stem from the interaction of multiple pathophysiological factors: ineffective erythropoiesis, iron overload (IOL), and hypercoagulability. Ineffective erythropoiesis and hemolysis are associated with chronic hypoxia and a hypercoagulable state. The latter are linked to a high prevalence of thromboembolic and cerebrovascular events, as well as leg ulcers and pulmonary hypertension. IOL in NTDT patients is a cumulative process that can lead to several iron-related morbidities in the liver (liver fibrosis), kidneys, endocrine glands (endocrinopathies), and vascular system (vascular disease). This review sheds light on the pathophysiology underlying morbidities associated with NTDT and summarizes the mainstays of treatment and some of the possible future therapeutic interventions.
Non-transfusion-dependent thalassemia: An update on complications and management / S. Joseph, T. Ali, B. Rayan, S. Antoine N., M.D. Cappellini, T. Ali T.. - In: INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. - ISSN 1661-6596. - 19:1(2018), pp. 182.1-182.16.
|Titolo:||Non-transfusion-dependent thalassemia: An update on complications and management|
CAPPELLINI, MARIA DOMENICA (Penultimo)
|Parole Chiave:||Ineffective erythropoiesis; Iron chelation therapy; Iron overload; Management; Morbidity; Non-transfusion dependent thalassemia; Catalysis; Molecular Biology; Spectroscopy; Computer Science Applications1707 Computer Vision and Pattern Recognition; Physical and Theoretical Chemistry; Organic Chemistry; Inorganic Chemistry|
|Settore Scientifico Disciplinare:||Settore MED/09 - Medicina Interna|
|Data di pubblicazione:||2018|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.3390/ijms19010182|
|Appare nelle tipologie:||01 - Articolo su periodico|