Efficacy and safety of deferasirox, an oral iron chelator, in heavily iron-overloaded patients with β-thalassaemia: the ESCALATOR study

Objective:  Many patients with transfusional iron overload are at risk for progressive organ dysfunction and early death and poor compliance with older chelation therapies is believed to be a major contributing factor. Phase II/III studies have shown that oral deferasirox 20–30 mg/kg/d reduces iron...

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Veröffentlicht in:European journal of haematology 2009-06, Vol.82 (6), p.458-465
Hauptverfasser: Taher, Ali, El-Beshlawy, Amal, Elalfy, Mohsen S., Al Zir, Kusai, Daar, Shahina, Habr, Dany, Kriemler-Krahn, Ulrike, Hmissi, Abdel, Al Jefri, Abdullah
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Sprache:eng
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Zusammenfassung:Objective:  Many patients with transfusional iron overload are at risk for progressive organ dysfunction and early death and poor compliance with older chelation therapies is believed to be a major contributing factor. Phase II/III studies have shown that oral deferasirox 20–30 mg/kg/d reduces iron burden, depending on transfusional iron intake. Methods:  The prospective, open‐label, 1‐yr ESCALATOR study in the Middle East was designed to evaluate once‐daily deferasirox in patients ≥2 yr with β‐thalassaemia major and iron overload who were previously chelated with deferoxamine and/or deferiprone. Most patients began treatment with deferasirox 20 mg/kg/d; doses were adjusted in response to markers of over‐ or under‐chelation. The primary endpoint was treatment success, defined as a reduction in liver iron concentration (LIC) of ≥3 mg Fe/g dry weight (dw) if baseline LIC was ≥10 mg Fe/g dw, or final LIC of 1–7 mg Fe/g dw for patients with baseline LIC of 2 to
ISSN:0902-4441
1600-0609
DOI:10.1111/j.1600-0609.2009.01228.x