title: Limitations of serum ferritin to predict liver iron concentration responses to deferasirox therapy in patients with transfusion-dependent thalassaemia.
abstract: BACKGROUND
  In transfusion-dependent anaemias, while absolute serum ferritin levels broadly correlate with liver iron concentration (LIC), relationships between trends in these variables are unclear. These relationships are important because serum ferritin changes are often used to adjust or switch chelation regimens when liver magnetic resonance imaging (MRI) is unavailable.
  
  
  OBJECTIVE
  This post hoc analysis of the EPIC study compared serum ferritin and LIC in 317 patients with transfusion-dependent thalassaemia before and after 1 yr of deferasirox.
  
  
  RESULTS
  Serum ferritin responses (decreases) occurred in 73% of patients, 80% of whom also have decreased LIC. However, 52% of patients without a serum ferritin response did decrease LIC and by >1 mg Fe/g dw (median 3.9) in 77% of cases. Absolute serum ferritin and LIC values correlated significantly only when serum ferritin was <4000 ng/mL (r = 0.59; P < 0.0001) and not at higher levels (≥4000 ng/mL; r = 0.19). Serum ferritin response was accompanied by decreased LIC in 89% and 70% of cases when serum ferritin was <4000 or ≥4000 ng/mL, respectively.
  
  
  CONCLUSIONS
  As serum ferritin non-response was associated with LIC decrease in over half of patients, use of liver MRI may be particularly useful for differentiating true from apparent non-responders to deferasirox based on serum ferritin trends alone.
authors.affiliation: Department of Haematology, University College London, London, UK.
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
authors.affiliation: Thalassemia Center, Children's Hospital, Ain Shams University, Cairo, Egypt.
authors.forename: Mohsen
authors.initials: M
authors.lastname: Elalfy
authors.affiliation: Department of Internal Medicine, American University of Beirut, Beirut, Lebanon.
authors.forename: Ali
authors.initials: A
authors.lastname: Taher
authors.affiliation: Department of Pediatric Hematology, Ege University Hospital, Izmir, Turkey.
authors.forename: Yesim
authors.initials: Y
authors.lastname: Aydinok
authors.affiliation: Department of Haematology, St George Hospital, Sydney, NSW, Australia.
authors.forename: Szu-Hee
authors.initials: SH
authors.lastname: Lee
authors.affiliation: Division of Haematology, Department of Medicine, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital, Bangkok, Thailand.
authors.forename: Pranee
authors.initials: P
authors.lastname: Sutcharitchan
authors.affiliation: Novartis Pharma AG, Basel, Switzerland.
authors.forename: Ali
authors.initials: A
authors.lastname: El-Ali
authors.affiliation: Novartis Pharmaceuticals, East Hanover, NJ, USA.
authors.forename: Jackie
authors.initials: J
authors.lastname: Han
authors.affiliation: Hematology Department, Pediatric Hospital, Cairo University, Cairo, Egypt.
authors.forename: Amal
authors.initials: A
authors.lastname: El-Beshlawy
mesh_terms: D000293:Adolescent; D000328:Adult; D001565:Benzoates / Q000627:therapeutic use*; D015415:Biomarkers; D015913:Chelation Therapy; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood*; D006801:Humans; D007501:Iron / Q000378:metabolism*; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology*; D008099:Liver / Q000378:metabolism*; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D012372:ROC Curve; D013789:Thalassemia / Q000097:blood* / Q000150:complications* / Q000628:therapy; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D001565:Benzoates; D015415:Biomarkers; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox
keywords: chelation; deferasirox; liver iron concentration; serum ferritin; thalassaemia