Management of non-transfusion-dependent thalassemia: a practical guide.

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.

• Authors: Ali T Taher, Maria Domenica Cappellini
• Journal: Drugs (Drugs) Vol. 74 Issue 15 Pg. 1719-29 (Oct 2014) ISSN: 1179-1950 [Electronic] New Zealand
• PMID: 25255924 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)

Chemical References

• Iron Chelating Agents

Topics

• Chelation Therapy
• Humans
• Iron Chelating Agents (therapeutic use)
• Iron Overload
• Thalassemia (drug therapy)