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Review
. 2018 Nov 1;10(1):e2018066.
doi: 10.4084/MJHID.2018.066. eCollection 2018.

Deferasirox: Over a Decade of Experience in Thalassemia

Nour M Moukalled  1 , Rayan Bou-Fakhredin  1 , Ali T Taher  1
Affiliations
Review

Deferasirox: Over a Decade of Experience in Thalassemia

Nour M Moukalled et al. Mediterr J Hematol Infect Dis. .

Abstract

Thalassemia incorporates a broad clinical spectrum characterized by decreased or absent production of normal hemoglobin leading to decreased red blood cell survival and ineffective erythropoiesis. Chronic iron overload remains an inevitable complication resulting from regular blood transfusions (transfusion-dependent) and/or increased iron absorption (mainly non-transfusion-dependent thalassemia), requiring adequate treatment to prevent the significant associated morbidity and mortality. Iron chelation therapy has become a cornerstone in the management of thalassemia patients, leading to improvements in their outcome and quality of life. Deferasirox (DFX), an oral iron chelating agent, is approved for use in transfusion dependent and non-transfusion-dependent thalassemia and has shown excellent efficacy in this setting. We herein present an updated review of the role of deferasirox in thalassemia, exploring over a decade of experience, which has documented its effectiveness and convenience; in addition to its manageable safety profile.

Keywords: Deferasirox; Iron chelation therapy; Iron overload; Liver iron concentration; Non-transfusion dependent thalassemia; Serum ferritin; Transfusion dependent thalassemia.

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Conflict of interest statement

Competing interests: The authors have declared that no competing interests exist.

Figures

Figure 1A
Figure 1A. DFX in thalassemia
DFX: deferasirox; TDT: transfusion-dependent thalassemia; NTDT: non-transfusion-dependent thalassemia; pRBCs: packed red blood cells; SF: serum ferritin; LIC: liver iron concentration; g dw: gram dry weight; DFO: deferoxamine; MRI: magnetic resonance imaging; Screa: serum creatinine; BL: baseline Note that the DFX doses provided are for the dispersible formulation, for the film coated tablet the dosage is around 30% less and the conversion used is:

  1. - 10 mg/kg/day DFX dispersible formula image 7 mg/kg/day FCT

  2. - 20 mg/kg/day DFX dispersible formula image 14 mg/kg/day FCT

  3. - 40 mg/kg/day DFX dispersible formula image 28 mg/kg/day FCT

Figure 1B
Figure 1B. Monitoring for and managing adverse events with DFX
AEs: adverse events; DFX: deferasirox; BL: baseline; Screa: serum creatinine.
See this image and copyright information in PMC

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