Document Detail

Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia.
MedLine Citation:
PMID:  19996412     Owner:  NLM     Status:  MEDLINE    
Cardiac iron overload causes most deaths in beta-thalassemia major. The efficacy of deferasirox in reducing or preventing cardiac iron overload was assessed in 192 patients with beta-thalassemia in a 1-year prospective, multicenter study. The cardiac iron reduction arm (n = 114) included patients with magnetic resonance myocardial T2* from 5 to 20 ms (indicating cardiac siderosis), left ventricular ejection fraction (LVEF) of 56% or more, serum ferritin more than 2500 ng/mL, liver iron concentration more than 10 mg Fe/g dry weight, and more than 50 transfused blood units. The prevention arm (n = 78) included otherwise eligible patients whose myocardial T2* was 20 ms or more. The primary end point was the change in myocardial T2* at 1 year. In the cardiac iron reduction arm, the mean deferasirox dose was 32.6 mg/kg per day. Myocardial T2* (geometric mean +/- coefficient of variation) improved from a baseline of 11.2 ms (+/- 40.5%) to 12.9 ms (+/- 49.5%) (+16%; P < .001). LVEF (mean +/- SD) was unchanged: 67.4 (+/- 5.7%) to 67.0 (+/- 6.0%) (-0.3%; P = .53). In the prevention arm, baseline myocardial T2* was unchanged from baseline of 32.0 ms (+/- 25.6%) to 32.5 ms (+/- 25.1%) (+2%; P = .57) and LVEF increased from baseline 67.7 (+/- 4.7%) to 69.6 (+/- 4.5%) (+1.8%; P < .001). This prospective study shows that deferasirox is effective in removing and preventing myocardial iron accumulation. This study is registered at as NCT00171821.
Dudley J Pennell; John B Porter; Maria Domenica Cappellini; Amal El-Beshlawy; Lee Lee Chan; Yesim Aydinok; Mohsen Saleh Elalfy; Pranee Sutcharitchan; Chi-Kong Li; Hishamshah Ibrahim; Vip Viprakasit; Antonis Kattamis; Gillian Smith; Dany Habr; Gabor Domokos; Bernard Roubert; Ali Taher
Publication Detail:
Type:  Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't     Date:  2009-12-08
Journal Detail:
Title:  Blood     Volume:  115     ISSN:  1528-0020     ISO Abbreviation:  Blood     Publication Date:  2010 Mar 
Date Detail:
Created Date:  2010-03-26     Completed Date:  2010-04-13     Revised Date:  2014-02-19    
Medline Journal Info:
Nlm Unique ID:  7603509     Medline TA:  Blood     Country:  United States    
Other Details:
Languages:  eng     Pagination:  2364-71     Citation Subset:  AIM; IM    
Data Bank Information
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MeSH Terms
Benzoates / administration & dosage*,  adverse effects
Dose-Response Relationship, Drug
Ferritins / blood
Heart Failure / drug therapy,  etiology,  prevention & control*
Iron / metabolism
Iron Chelating Agents / administration & dosage*,  adverse effects
Iron Overload / drug therapy,  etiology,  prevention & control*
Myocardium / metabolism
Prospective Studies
Triazoles / administration & dosage*,  adverse effects
Young Adult
beta-Thalassemia / complications,  drug therapy*
Grant Support
PG/09/074/27961//British Heart Foundation
Reg. No./Substance:
0/Benzoates; 0/Iron Chelating Agents; 0/Triazoles; 9007-73-2/Ferritins; E1UOL152H7/Iron; V8G4MOF2V9/deferasirox
Comment In:
Blood. 2010 Mar 25;115(12):2333-4   [PMID:  20339104 ]

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