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Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease.

Author(s): Vichinsky E, Bernaudin F, Forni GL, Gardner R, Hassell K, Heeney MM, Inusa B, Kutlar A, Lane P, Mathias L, Porter J, Tebbi C, Wilson F, Griffel L, Deng W, Giannone V, Coates T

Affiliation(s): Children's Hospital and Research Center at Oakland, Oakland, CA 94609, USA. evichinsky@mail.cho.org

Publication date & source: 2011-08, Br J Haematol., 154(3):387-97. Epub 2011 May 19.

Publication type: Clinical Trial, Phase II; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't

To date, there is a lack of long-term safety and efficacy data for iron chelation therapy in transfusion-dependent patients with sickle cell disease (SCD). To evaluate the long-term safety and efficacy of deferasirox (a once-daily oral iron chelator), patients with SCD completing a 1-year, Phase II, randomized, deferoxamine (DFO)-controlled study entered a 4-year extension, continuing to receive deferasirox, or switching from DFO to deferasirox. Average actual deferasirox dose was 19.4 +/- 6.3 mg/kg per d. Of 185 patients who received at least one deferasirox dose, 33.5% completed the 5-year study. The most common reasons for discontinuation were withdrawal of consent (23.8%), lost to follow-up (9.2%) and adverse events (AEs) (7.6%). Investigator-assessed drug-related AEs were predominantly gastrointestinal [including nausea (14.6%), diarrhoea (10.8%)], mild-to-moderate and transient in nature. Creatinine clearance remained within the normal range throughout the study. Despite conservative initial dosing, serum ferritin levels in patients with >/= 4 years deferasirox exposure significantly decreased by -591 mug/l (95% confidence intervals, -1411, -280 mug/l; P = 0.027; n = 67). Long-term deferasirox treatment for up to 5 years had a clinically acceptable safety profile, including maintenance of normal renal function, in patients with SCD. Iron burden was substantially reduced with appropriate dosing in patients treated for at least 4 years. (c) 2011 Blackwell Publishing Ltd.

Page last updated: 2011-12-09

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