Deferasirox for the treatment of iron overload in non-transfusion-dependent thalassemia

Expert Review of Hematology
Ali TaherMaria Domenica Cappellini

Abstract

Non-transfusion-dependent thalassemia (NTDT) defines a group of patients who do not require regular transfusions for survival, but are at significant risk of iron accumulation from underlying disease-related mechanisms distinct from transfusional iron overload. Management of iron overload in NTDT has received little attention compared with that of β-thalassemia major, despite evidence of significant iron-induced complications with advancing age. The efficacy and safety of the iron chelator deferasirox in NTDT has been evaluated in two pilot studies and the first prospective, randomized, placebo-controlled study (THALASSA) of any chelator in NTDT. Treatment with deferasirox for up to 2 years yielded a sustained reduction in iron burden, with a clinically manageable safety profile. Following these trial data, deferasirox is the first iron chelator approved for use in NTDT patients, and with NTDT guidelines now available, physicians are better equipped to achieve effective monitoring and management of iron burden in NTDT.

References

Nov 1, 1981·European Journal of Pediatrics·P CossuAntonio Cao
Sep 1, 1995·Annals of Hematology·T LombardoV Calvi
Aug 24, 2000·The New England Journal of Medicine·F E ChenVivian Chan
Jul 9, 2003·British Journal of Haematology·Pensri PootrakulA V Hoffbrand
Apr 9, 2004·Lancet·Y GandonYves Deugnier
Aug 26, 2004·British Journal of Haematology·Ersi VoskaridouDimitris Loukopoulos
Dec 13, 2005·Annals of the New York Academy of Sciences·Elliott P Vichinsky
Apr 14, 2006·British Journal of Haematology·Joyce C W ChanVivian Chan
Jun 2, 2006·Blood Cells, Molecules & Diseases·Ali TaherMaria Domenica Cappellini
Apr 7, 2007·Current Opinion in Hematology·John C Wood
May 10, 2007·Haematologica·Raffaella OrigaElizabeta Nemeth
Nov 21, 2007·Hematology·Elliott P Vichinsky
Dec 7, 2007·American Journal of Hematology·Elliott P Vichinsky
Sep 9, 2008·Drug Metabolism and Disposition : the Biological Fate of Chemicals·Gerard J BruinFelix Waldmeier
Dec 9, 2008·Pediatric Blood & Cancer·Jennifer MichlitschElliott P Vichinsky
Mar 7, 2009·Blood·Jane S HankinsClaudia M Hillenbrand
Apr 25, 2009·Annals of Hematology·Vichai LaosombatBenjamas Sattayasevana
Jul 28, 2009·Annual Review of Medicine·Maria Domenica Cappellini, Paolo Pattoneri
Aug 18, 2009·British Journal of Haematology·Ali TaherMaria Domenica Cappellini
Dec 17, 2009·Hematology·Roland Fischer, Paul Harmatz
Jan 26, 2010·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Antonio Cao, Renzo Galanello
May 25, 2010·Orphanet Journal of Rare Diseases·Renzo Galanello, Raffaella Origa
May 29, 2010·Orphanet Journal of Rare Diseases·Cornelis L Harteveld, Douglas R Higgs
Nov 19, 2010·Expert Review of Hematology·Roshan ColahAnita Nadkarni
Jan 22, 2011·British Journal of Haematology·Ali TaherDavid J Weatherall
Feb 25, 2011·The New England Journal of Medicine·Ashutosh LalElliott P Vichinsky
Mar 23, 2011·Hematology·Krittapoom AkrawinthawongNoppadol Siritanaratkul
Apr 1, 2011·Expert Opinion on Therapeutic Patents·Hossein HeliKhashayar Karimian
Dec 20, 2011·Expert Opinion on Drug Metabolism & Toxicology·Renzo GalanelloRaffaella Origa
Mar 30, 2012·Current Medicinal Chemistry·Y MaR C Hider
May 29, 2012·Blood Reviews·Khaled M MusallamAli Taher
May 29, 2012·Blood Reviews·Elliott P Vichinsky
Jul 5, 2012·Cold Spring Harbor Perspectives in Medicine·Khaled M MusallamEliezer A Rachmilewitz
Mar 12, 2013·American Journal of Hematology·Ali TaherMaria Domenica Cappellini

Citations

Feb 26, 2010·BMC Blood Disorders·Montarat ThavorncharoensapBang-On Ubol
Jul 5, 2015·Blood Cells, Molecules & Diseases·Ali TaherMaria Domenica Cappellini
May 18, 2016·International Journal of Laboratory Hematology·V BrancaleoniMaria Domenica Cappellini
Oct 22, 2014·World Journal of Methodology·Christina N KontoghiorgheGeorge J Kontoghiorghes
Oct 17, 2019·Revista da Associação Médica Brasileira·Tadeu Gonçalves de LimaAna Rosa Pinto Quidute
Aug 16, 2017·The Cochrane Database of Systematic Reviews·Claudia BolligJoerg J Meerpohl

Related Concepts

Deferasirox
Benzoate
Blood Transfusion
Iron Chelating Agents
Thalassemia
Triazoles
Iron Overload
Blood Transfusion
Chelating Agents
Clinical Trials

Related Feeds

Anemia

Anemia develops when your blood lacks enough healthy red blood cells. Anemia of inflammation (AI, also called anemia of chronic disease) is a common, typically normocytic, normochromic anemia that is caused by an underlying inflammatory disease. Here is the latest research on anemia.