期刊
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
卷 19, 期 1, 页码 -出版社
MDPI
DOI: 10.3390/ijms19010182
关键词
non-transfusion dependent thalassemia; morbidity; management; ineffective erythropoiesis; iron overload; iron chelation therapy
资金
- Novartis Pharmaceuticals
- Celgene
- Roche
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.
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