Yazarlar : Taher A, Elalfy MS, Al Zir K, Daar S, Al Jefri A, Habr D, Kriemler-Krahn U, El-Ali A, Roubert B, El-Beshlawy A.

Yayın : Eur J Haematol.

Yayın Yılı : 2011

Pubmed Linki : http://www.ncbi.nlm.nih.gov/pubmed/21668502

Konu : Talasemi

Literatür İçeriği :

Abstract

Following 1 year's deferasirox therapy in the ESCALATOR study, 57% of previously chelated patients with β-thalassaemia achieved treatment success (maintenance/reduction of liver iron concentration [LIC] versus baseline LIC). 78% had dose increases at median of 26 wk, suggesting that 1-year results may not have reflected full deferasirox efficacy. Extension data are presented here. Deferasirox starting dose was 20 mg/kg/d (increases to 30/40 mg/kg/d permitted in the core/extension, respectively). Efficacy was primarily assessed by absolute change in LIC and serum ferritin. Overall, 231 patients received deferasirox in the extension; 67.4% (P < 0.0001) achieved treatment success. 66.2% of patients were receiving doses ≥30 mg/kg/d by the end of the extension. By the end of the 1-year extension, mean LIC had decreased by 6.6 ± 9.4 mg Fe/g dw (baseline 19.6 ± 9.2; P < 0.001) and median serum ferritin by 929 ng/mL (baseline 3356; P < 0.0001). There was a concomitant improvement in liver function markers (P < 0.0001). Fewer drug-related adverse events were reported in extension than core study (23.8% vs 44.3%). Doses ≥30 mg/kg/d were generally required because of high transfusional iron intake and high baseline serum ferritin levels, highlighting the importance of administering an adequate dose to achieve net negative iron balance 

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