Management of non-transfusion-dependent thalassemia: A practical guide

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Despite their transfusion-independence, non-transfusion-dependent thalassemia (NTDT) patients experience a variety of serious clinical complications that require prompt and comprehensive management. Transfusion therapy may still be an important part of management of this disease, in cases of acute stress, to support growth and development in childhood, or to prevent clinical morbidities stemming from ineffective erythropoiesis or hemolytic anemia. Although splenectomy is associated with improvements in hemoglobin levels, it leads to several short- and long-term adverse events, warranting caution in application of this intervention. Fetal hemoglobin induction therapy has been evaluated in non-randomized studies, with benefits extending beyond hematologic improvements to lowering morbidity risk. Effective and safe iron chelation therapy is now available for NTDT patients in whom iron overload develops, irrespective of transfusions, due to increased intestinal absorption, ultimately leading to clinically high iron burden levels and subsequent morbidity. Optimal management of NTDT patients requires a holistic approach targeting all hallmarks of the disease to ensure favorable patient outcomes.

Original languageEnglish
Pages (from-to)1719-1729
Number of pages11
JournalDrugs
Volume74
Issue number15
DOIs
Publication statusPublished - Sep 26 2014

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Thalassemia
Morbidity
Iron
Chelation Therapy
Fetal Hemoglobin
Iron Overload
Erythropoiesis
Hemolytic Anemia
Intestinal Absorption
Splenectomy
Disease Management
Growth and Development
Hemoglobins
Therapeutics

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Medicine(all)

Cite this

Management of non-transfusion-dependent thalassemia : A practical guide. / Taher, Ali T.; Cappellini, Maria Domenica.

In: Drugs, Vol. 74, No. 15, 26.09.2014, p. 1719-1729.

Research output: Contribution to journalArticle

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