Τίτλος:
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
Γλώσσες Τεκμηρίου:
Αγγλικά
Περίληψη:
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.
Συγγραφείς:
Taher, A.T.
Porter, J.B.
Viprakasit, V.
Kattamis, A.
Chuncharunee, S.
Sutcharitchan, P.
Siritanaratkul, N.
Origa, R.
Karakas, Z.
Habr, D.
Zhu, Z.
Cappellini, M.D.
Περιοδικό:
British Journal of Haematology
Εκδότης:
Wiley-Blackwell Publishing Ltd
Λέξεις-κλειδιά:
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
