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Response of iron overload to deferasirox in rare transfusion-dependent anaemias: equivalent effects on serum ferritin and labile plasma iron for haemolytic or production anaemias.
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 transfusiondependent
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) 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 < 0.0001) and haemolytic (P = 0.037) anaemias after
the first dose and was maintained at normal mean levels (<0.4 lm) subsequently. The most common
drug-related, investigator-assessed adverse events were diarrhoea (n = 16) and nausea (n = 12). Conclusions:
At matched transfusional iron-loading rates, the responses of rare transfusion-dependent anaemias
to deferasirox are similar at 1 yr, irrespective of the underlying pathogenic mechanism
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