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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
medicine
Importance of optimal dosing ≥30mg/kg/d during deferasirox treatment: 2.7-yr follow-up from the ESCALATOR study in patients with β-thalassaemia
European Journal of Haematology, Volume 87, No. 4, Year 2011
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Description
Following 1-yr deferasirox therapy in the ESCALATOR study, 57% of previously chelated patients with β-thalassaemia achieved treatment success (maintenance of or reduction in liver iron concentration (LIC) vs. baseline LIC). Seventy-eight per cent had dose increases at median of 26wk, suggesting that 1-yr results may not have reflected full deferasirox efficacy. Extension data are presented here. Deferasirox starting dose was 20mg/kg/d (increases to 30/40mg/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. By the end of the extension, 66.2% of patients were receiving doses ≥30mg/kg/d. By the end of the 1-yr extension, mean LIC had decreased by 6.6±9.4mg Fe/g dw (baseline 19.6±9.2; P<0.001) and median serum ferritin by 929ng/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 ≥30mg/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. © 2011 John Wiley & Sons A/S.
Authors & Co-Authors
Taher, Ali T.
Lebanon, Beirut
American University of Beirut Medical Center
ElAlfy, Mohsen Saleh
Egypt, Cairo
Ain Shams University
Al-Zir, Kusai Nasser
Syrian Arab Republic, Damascus
National Thalassemia Center
Daar, Shahina F.
Oman, Muscat
Sultan Qaboos University
Al-Jefri, Abdullah Hussain
Saudi Arabia, Riyadh
King Faisal Specialist Hospital and Research Centre
Habr, Dany
Switzerland, Basel
Novartis International ag
Kriemler-Krahn, Ulrike
Switzerland, Basel
Novartis International ag
El-Ali, Ali
Switzerland, Basel
Novartis International ag
Roubert, Bernard
Switzerland, Basel
Novartis International ag
El-Beshlawy, Amal M.
Egypt, Giza
Cairo University
Statistics
Citations: 36
Authors: 10
Affiliations: 7
Identifiers
Doi:
10.1111/j.1600-0609.2011.01662.x
ISSN:
09024441
e-ISSN:
16000609
Study Design
Cohort Study