Non-transfusion-dependent thalassemia: An update on complications and management

Joseph Sleiman, Ali Tarhini, Rayan Bou-Fakhredin, Antoine N. Saliba, Maria Domenica Cappellini, Ali T. Taher

Research output: Contribution to journalReview article

Abstract

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.

Original languageEnglish
Article number182
JournalInternational Journal of Molecular Sciences
Volume19
Issue number1
DOIs
Publication statusPublished - Jan 8 2018

Keywords

  • Ineffective erythropoiesis
  • Iron chelation therapy
  • Iron overload
  • Management
  • Morbidity
  • Non-transfusion dependent thalassemia

ASJC Scopus subject areas

  • Catalysis
  • Molecular Biology
  • Spectroscopy
  • Computer Science Applications
  • Physical and Theoretical Chemistry
  • Organic Chemistry
  • Inorganic Chemistry

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