Wiley, European Journal of Haematology, 4(87), p. 338-348, 2011
DOI: 10.1111/j.1600-0609.2011.01660.x
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Objectives: It is widely assumed that, at matched transfusional iron- loading rates, responses to chelation therapy are similar, irrespective of the underlying condition. However, data are limited for rare transfusion- dependent anaemias, and it remains to be elucidated if response differs, depending on whether the anaemia has a primary haemolytic or production mechanism. Methods: The efficacy and safety of deferasirox (Exjade (R)) in rare transfusion-dependent anaemias were evaluated over 1 yr, with change in serum ferritin as the primary efficacy endpoint. Initial deferasirox doses were 10-30 mg/kg/d, depending on transfusion requirements; 34 patients had production anaemias, and 23 had haemolytic anaemias. Results : Patients with production anaemias or haemolytic anaemias had comparable transfusional iron-loading rates (0.31 vs. 0. 30 mL red blood cells/kg/d), mean deferasirox dosing (19.3 vs. 19.0 mg/kg/d) and baseline median serum ferritin (2926 vs. 2682 ng/mL). Baseline labile plasma iron (LPI) levels correlated significantly with the transfusional iron-loading rates and with serum ferritin levels in both cohorts. Reductions in median serum ferritin levels were initially faster in the production than the haemolytic anaemias, but at 1 yr, similar significant reductions of 940 and 617 ng/mL were attained, respectively (-26.0% overall). Mean LPI decreased significantly in patients with production (P