title: Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort.
abstract: Morbidity and mortality in thalassemia are associated with iron burden. Recent advances in organ-specific iron imaging and the availability of oral deferasirox are expected to improve clinical care, but the extent of use of these resources and current chelation practices have not been well described. In the present study, we studied chelation use and the change in iron measurements in 327 subjects with transfusion-dependent thalassemia (mean entry age, 22.1 ± 2.5 years) from 2002-2011, with a mean follow-up of 8.0 years (range, 4.4-9.0 years). The predominant chelator currently used is deferasirox, followed by deferoxamine and then combination therapies. The use of both hepatic and cardiac magnetic resonance imaging increased more than 5-fold (P < .001) during the study period, leading to an 80% increase in the number of subjects undergoing liver iron concentration (LIC) measurements. Overall, LIC significantly improved (median, 10.7 to 5.1 mg/g dry weight, P < .001) with a nonsignificant improvement in cardiac T2* (median, 23.55 to 34.50 ms, P = .23). The percentage of patients with markers of inadequate chelation (ferritin > 2500 ng/mL, LIC > 15 mg/g dry weight, and/or cardiac T2* < 10 ms) also declined from 33% to 26%. In summary, increasing use of magnetic resonance imaging and oral chelation in thalassemia management has likely contributed to improved iron burden.
authors.affiliation: Children's Hospital of Philadelphia and University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA. kwiatkowski@e-mail.chop.edu
authors.forename: Janet L
authors.initials: JL
authors.lastname: Kwiatkowski
authors.forename: Hae-Young
authors.initials: HY
authors.lastname: Kim
authors.forename: Alexis A
authors.initials: AA
authors.lastname: Thompson
authors.forename: Charles T
authors.initials: CT
authors.lastname: Quinn
authors.forename: Brigitta U
authors.initials: BU
authors.lastname: Mueller
authors.forename: Isaac
authors.initials: I
authors.lastname: Odame
authors.forename: Patricia J
authors.initials: PJ
authors.lastname: Giardina
authors.forename: Elliott P
authors.initials: EP
authors.lastname: Vichinsky
authors.forename: Jeanne M
authors.initials: JM
authors.lastname: Boudreaux
authors.forename: Alan R
authors.initials: AR
authors.lastname: Cohen
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
authors.forename: Thomas
authors.initials: T
authors.lastname: Coates
authors.forename: Nancy F
authors.initials: NF
authors.lastname: Olivieri
authors.forename: Ellis J
authors.initials: EJ
authors.lastname: Neufeld
mesh_terms: D015331:Cohort Studies; D005260:Female; D006801:Humans; D007501:Iron / Q000032:analysis*; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008099:Liver / Q000737:chemistry* / Q000378:metabolism; D008131:London; D008137:Longitudinal Studies; D008279:Magnetic Resonance Imaging; D008297:Male; D009656:North America; D013789:Thalassemia / Q000188:drug therapy* / Q000378:metabolism; D055815:Young Adult
chemical_list: D007502:Iron Chelating Agents; D007501:Iron