Safety profiles of iron chelators in young patients with haemoglobinopathies
Botzenhardt S, Li N, Chan EW, Sing CW, Wong ICK, Neubert A (2017)
Publication Type: Journal article
Publication year: 2017
Journal
Book Volume: 98
Pages Range: 198-217
Journal Issue: 3
DOI: 10.1111/ejh.12833
Abstract
BACKGROUND: This review describes the safety of deferoxamine (DFO), deferiprone (DFP), deferasirox (DFX) and combined therapy in young patients less than 25 yr of age with haemoglobinopathies. METHODS: 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 2040 patients from 34 studies were included. Ninety-two 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 correspond 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 are scarce, but it seems equally safe compared to monotherapy.
Authors with CRIS profile
Sebastian Botzenhardt
Department of Paediatrics and Adolescent Medicine
Antje Neubert
Department of Paediatrics and Adolescent Medicine
Involved external institutions
Hong Kong (HK)How to cite
APA:
Botzenhardt, S., Li, N., Chan, E.W., Sing, C.W., Wong, I.C.K., & Neubert, A. (2017). Safety profiles of iron chelators in young patients with haemoglobinopathies. European Journal of Haematology, 98(3), 198-217. https://doi.org/10.1111/ejh.12833
MLA:
Botzenhardt, Sebastian, et al. "Safety profiles of iron chelators in young patients with haemoglobinopathies." European Journal of Haematology 98.3 (2017): 198-217.
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