TY - JOUR
TI - 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
AU - Taher, A.T.
AU - Porter, J.B.
AU - Viprakasit, V.
AU - Kattamis, A.
AU - Chuncharunee, S.
AU - Sutcharitchan, P.
AU - Siritanaratkul, N.
AU - Origa, R.
AU - Karakas, Z.
AU - Habr, D.
AU - Zhu, Z.
AU - Cappellini, M.D.
JO - British Journal of Haematology
PY - 2015
VL - 168
TODO - 2
SP - 284-290
PB - Wiley-Blackwell Publishing Ltd
SN - 0007-1048, 1365-2141
TODO - 10.1111/bjh.13119
TODO - deferasirox;  ferritin;  iron;  placebo;  benzoic acid derivative;  deferasirox;  ferritin;  iron;  iron chelating agent;  triazole derivative, Article;  chelation;  concentration response;  controlled study;  correlation analysis;  double blind procedure;  drug efficacy;  drug safety;  ferritin blood level;  human;  iron overload;  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;  thalassemia;  adolescent;  adult;  blood;  clinical trial;  female;  liver;  male;  metabolism;  middle aged;  nuclear magnetic resonance imaging;  thalassemia;  young adult, Adolescent;  Adult;  Benzoates;  Double-Blind Method;  Female;  Ferritins;  Humans;  Iron;  Iron Chelating Agents;  Liver;  Magnetic Resonance Imaging;  Male;  Middle Aged;  Thalassemia;  Triazoles;  Young Adult
TODO - 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.
ER -