Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non-transfusion-dependent thalassaemia
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Blackwell Publishing Ltd
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Summary: Liver iron concentration (LIC) assessment by magnetic resonance imaging (MRI) remains the gold standard to diagnose iron overload and guide iron chelation therapy in patients with non-transfusion-dependent thalassaemia (NTDT). However, limited access to MRI technology and expertise worldwide makes it practical to also use serum ferritin assessments. The THALASSA (assessment of Exjade® in non-transfusion-dependent THALASSemiA patients) study assessed the efficacy and safety of deferasirox in iron-overloaded NTDT patients and provided a large data set to allow exploration of the relationship between LIC and serum ferritin. Using data from screened patients and those treated with deferasirox for up to 2 years, we identified clinically relevant serum ferritin thresholds (for when MRI is unavailable) for the initiation of chelation therapy (>800 μg/l), as well as thresholds to guide chelator dose interruption (<300 μg/l) and dose escalation (>2000 μg/l). (clinicaltrials.gov identifier: NCT00873041). © 2014 The Authors. British Journal of Haematology published by John Wiley & Sons Ltd.
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Chelation therapy, Iron overload, Liver iron concentration, Non-transfusion-dependent thalassaemia, Serum ferritin, Adolescent, Adult, Benzoates, Double-blind method, Female, Ferritins, Humans, Iron, Iron chelating agents, Liver, Magnetic resonance imaging, Male, Middle aged, Thalassemia, Triazoles, Young adult, Deferasirox, Ferritin, Placebo, Benzoic acid derivative, Iron chelating agent, Triazole derivative, Article, Chelation, Concentration response, Controlled study, Correlation analysis, Double blind procedure, Drug efficacy, Drug safety, Ferritin blood level, Human, Liver level, Major clinical study, Multicenter study, Non transfusion dependent thalassemia, Nuclear magnetic resonance imaging, Nuclear magnetic resonance scanner, Phase 2 clinical trial, Predictive value, Priority journal, Randomized controlled trial, Receiver operating characteristic, Sensitivity and specificity, Blood, Clinical trial, Metabolism