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
Journal
European Journal of Haematology
Journal Volume
87
Journal Issue
4
Pages
338-348
Date Issued
2011
Author(s)
Abstract
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 ?) in rare transfusion-dependent anaemias were evaluated over 1yr, with change in serum ferritin as the primary efficacy endpoint. Initial deferasirox doses were 10-30mg/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. 0.30mLred blood cells/kg/d), mean deferasirox dosing (19.3 vs. 19.0mg/kg/d) and baseline median serum ferritin (2926 vs. 2682ng/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 1yr, similar significant reductions of 940 and 617ng/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 (<0.4μm) 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 1yr, irrespective of the underlying pathogenic mechanism. ? 2011 John Wiley & Sons A/S.
Subjects
Iron chelation therapy; Iron overload; Rare anaemias; Safety; Serum ferritin
SDGs
Other Subjects
cyclosporin; deferasirox; deferoxamine; ferritin; iron; abdominal distension; adolescent; alanine aminotransferase blood level; anemia; angioneurotic edema; article; chelation therapy; child; cholelithiasis; clinical trial; constipation; creatinine blood level; diarrhea; dizziness; drug dose reduction; drug efficacy; drug response; drug safety; drug tolerability; drug withdrawal; dyspepsia; erythrocyte transfusion; fatigue; female; ferritin blood level; hemolytic anemia; human; hyperpyrexia; iron blood level; iron chelation; iron overload; major clinical study; male; multicenter study; nausea; open study; patient compliance; preschool child; priority journal; prospective study; pruritus; pure red cell anemia; rash; rebound; school child; side effect; transfusion dependent anemia; upper abdominal pain; vomiting; Adolescent; Anemia; Benzoic Acids; Blood Transfusion; Child; Child, Preschool; Female; Ferritins; Humans; Iron; Iron Chelating Agents; Iron Overload; Male; Triazoles
Type
journal article