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

Ali T. Taher, Sally Temraz, M. Domenica Cappellini

Research output: Contribution to journalArticle

6 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)495-509
Number of pages15
JournalExpert Review of Hematology
Volume6
Issue number5
DOIs
Publication statusPublished - 2013

Fingerprint

Iron Overload
Thalassemia
Iron
Chelating Agents
Therapeutics
Safety
beta-Thalassemia
deferasirox
Placebos
Guidelines
Physicians
Survival

Keywords

  • deferasirox
  • iron chelation
  • iron overload
  • thalassemia

ASJC Scopus subject areas

  • Hematology

Cite this

Deferasirox for the treatment of iron overload in non-transfusion-dependent thalassemia. / Taher, Ali T.; Temraz, Sally; Domenica Cappellini, M.

In: Expert Review of Hematology, Vol. 6, No. 5, 2013, p. 495-509.

Research output: Contribution to journalArticle

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