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Safety Profiles of Iron Chelators in Young Patients with Haemoglobinopathies

Botzenhardt, S; Li, N; Chan, EW; Sing, CW; Wong, IC; Neubert, A; (2017) Safety Profiles of Iron Chelators in Young Patients with Haemoglobinopathies. European Journal of Haematology , 98 (3) pp. 198-217. 10.1111/ejh.12833. Green open access

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Abstract

BACKGROUND: This review describes the safety of deferoxamine (DFO), deferiprone (DFP), deferasirox (DFX), and combined therapy in young patients less than 25 years of age with haemoglobinopathies. METHOD: Searches in electronic literature databases were performed. Studies reporting adverse events associated with iron chelation therapy were included. Study and reporting quality was assessed using AHRQ Risk of Bias Assessment Tool and McMaster Quality Assessment Scale of Harms. Prospective clinical studies were pooled in a random-effects meta-analysis of proportions. RESULTS: Safety data of 2,040 patients from 34 studies were included. 92 case reports of 246 patients were identified. DFX (937 patients) and DFP (667 patients) possess the largest published safety evidence. Fewer studies on combination regimens are available. Increased transaminases were seen in all regimens (3.9-31.3%) and gastrointestinal disorders with DFP and DFX (3.7-18.4% and 5.8-18.8%, respectively). Therapy discontinuations due to adverse events were low (0-4.1%). Reporting quality was selective and poor in most of the studies. CONCLUSION: Iron chelation therapy is generally safe in young patients and published data corresponds to summary of product characteristics. Each iron chelation regimen has its specific safety risks. DFO seems not to be associated with serious adverse effects in recommended doses. In DFP and DFX rare, but serious adverse reactions can occur. Data on combined therapy is scarce, but it seems equally safe compared to monotherapy. This article is protected by copyright. All rights reserved.

Type: Article
Title: Safety Profiles of Iron Chelators in Young Patients with Haemoglobinopathies
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/ejh.12833
Publisher version: http://doi.org/10.1111/ejh.12833
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Deferoxamine, adverse drug reactions, children, deferasirox, deferiprone, haemoglobinopathies
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 Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy > Practice and Policy
URI: http://discovery.ucl.ac.uk/id/eprint/1530975
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