An update on thalassemia intermedia

Joseph E. Maakaron, Maria Domenica Cappellini, Ali T. Taher

Research output: Contribution to journalArticle

Abstract

Thalassemia intermedia is a genetically diverse group of diseases that is the result of an imbalance in the production of the alpha and beta chains with ensuing chronic hemolysis, ineffective erythropoiesis, and iron overload. Resulting complications include bone changes, hypercoagulability, and end-organ damage due to iron overload. This decade has witnessed major breakthroughs in the management of thalassemia. In this article, we examine these novelties in therapy including iron chelation therapy, stem cell transplant, and gene therapy. Iron chelation therapy has been revolutionized with the advent of deferasirox, a once-daily oral iron chelator, that has been shown to be safe and efficacious. Gene therapy was also at the core of this revolution with the discovery of novel gene elements and viral vectors allowing for better control and improved outcomes.

Original languageEnglish
Pages (from-to)175-182
Number of pages8
JournalJournal Medical Libanais
Volume61
Issue number3
Publication statusPublished - 2013

Keywords

  • Gene therapy
  • Hematopoietic stem cell transplant
  • Hemoglobinopathy
  • Ineffective erythropoiesis
  • Iron chelation
  • Iron overload
  • Jak2 inhibitor
  • Thalassemia
  • Thalassemia intermedia

ASJC Scopus subject areas

  • Medicine(all)

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  • Cite this

    Maakaron, J. E., Cappellini, M. D., & Taher, A. T. (2013). An update on thalassemia intermedia. Journal Medical Libanais, 61(3), 175-182.