title: 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.
abstract: 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).
authors.affiliation: American University of Beirut, Beirut, Lebanon.
authors.forename: Ali T
authors.initials: AT
authors.lastname: Taher
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
authors.forename: Vip
authors.initials: V
authors.lastname: Viprakasit
authors.forename: Antonis
authors.initials: A
authors.lastname: Kattamis
authors.forename: Suporn
authors.initials: S
authors.lastname: Chuncharunee
authors.forename: Pranee
authors.initials: P
authors.lastname: Sutcharitchan
authors.forename: Noppadol
authors.initials: N
authors.lastname: Siritanaratkul
authors.forename: Raffaella
authors.initials: R
authors.lastname: Origa
authors.forename: Zeynep
authors.initials: Z
authors.lastname: Karakas
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Zewen
authors.initials: Z
authors.lastname: Zhu
authors.forename: Maria Domenica
authors.initials: MD
authors.lastname: Cappellini
mesh_terms: D000293:Adolescent; D000328:Adult; D001565:Benzoates / Q000008:administration & dosage*; D000077588:Deferasirox; D004311:Double-Blind Method; D005260:Female; D005293:Ferritins / Q000097:blood*; D006801:Humans; D007501:Iron / Q000378:metabolism*; D007502:Iron Chelating Agents / Q000008:administration & dosage*; D008099:Liver / Q000378:metabolism*; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D013789:Thalassemia / Q000097:blood / Q000188:drug therapy*; D014230:Triazoles / Q000008:administration & dosage*; D055815:Young Adult
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox
keywords: chelation therapy; iron overload; liver iron concentration; non-transfusion-dependent thalassaemia; serum ferritin