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Combined chelation therapy with deferasirox and deferoxamine in thalassemia

Lal, A; Porter, J; Sweeters, N; Ng, V; Evans, P; Neumayr, L; Kurio, G; ... Vichinsky, E; + view all (2013) Combined chelation therapy with deferasirox and deferoxamine in thalassemia. Blood Cells, Molecules, and Diseases , 50 (2) pp. 99-104. 10.1016/j.bcmd.2012.10.006.

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Abstract

Iron overload is the primary cause of mortality and morbidity in thalassemia major despite advances in chelation therapy. We performed a pilot clinical trial to evaluate the safety and efficacy of combined therapy with deferasirox (DFX, 20-30. mg/kg daily) and deferoxamine (DFO, 35-50. mg/kg on 3-7. days/week) in 22 patients with persistent iron overload or organ damage. In the 18 subjects completing 12. months of therapy, median liver iron concentration decreased by 31% from 17.4. mg/g (range 3.9-38.2. mg/g) to 12.0. mg/g (range 0.96-26.7 mg/g, p<0.001). Median ferritin decreased by 24% from 2465. ng/mL (range 1110-10,700. ng/mL) to 1875. ng/mL (range 421-5800. ng/mL, p=0.002). All 6 subjects with elevated myocardial iron showed improvement in MRI T2*(p=0.031). The mean±S.E. plasma non-transferrin-bound iron (NTBI) declined from 3.10±0.25 μM to 2.15±0.29 μM (p=0.028). The administration of DFX during infusion of DFO further lowered NTBI (-0.28±0.08 μM, p=0.004) and labile plasma iron (LPI, -0.03±0.01 μM, p=0.006). The simultaneous administration of DFO and DFX rapidly reduced systemic and myocardial iron, and provided an excellent control of the toxic labile plasma iron species without an increase in toxicity. This trial was registered at www.clinicaltrials.gov as NCT00901199. © 2012 Elsevier Inc.

Type: Article
Title: Combined chelation therapy with deferasirox and deferoxamine in thalassemia
DOI: 10.1016/j.bcmd.2012.10.006
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: http://discovery.ucl.ac.uk/id/eprint/1543525
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