See Appendix I for a complete list of investigators
A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease
Version of Record online: 21 DEC 2006
British Journal of Haematology
Volume 136, Issue 3, pages 501–508, February 2007
How to Cite
Vichinsky, E., Onyekwere, O., Porter, J., Swerdlow, P., Eckman, J., Lane, P., Files, B., Hassell, K., Kelly, P., Wilson, F., Bernaudin, F., Forni, G. L., Okpala, I., Ressayre-Djaffer, C., Alberti, D., Holland, J., Marks, P., Fung, E., Fischer, R., Mueller, B. U., Coates, T. and the Deferasirox in Sickle Cell Investigators (2007), A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. British Journal of Haematology, 136: 501–508. doi: 10.1111/j.1365-2141.2006.06455.x
Re-use of this article is permitted in accordance with the Creative Commons Deed, Attribution 2.5, which does not permit commercial exploitation.
- Issue online: 21 DEC 2006
- Version of Record online: 21 DEC 2006
- Received 27 September 2006; accepted for publication 13 November 2006
- sickle cell disease;
- iron overload
Deferasirox is a once-daily, oral iron chelator developed for treating transfusional iron overload. Preclinical studies indicated that the kidney was a potential target organ of toxicity. As patients with sickle cell disease often have abnormal baseline renal function, the primary objective of this randomised, open-label, phase II trial was to evaluate the safety and tolerability of deferasirox in comparison with deferoxamine in this population. Assessment of efficacy, as measured by change in liver iron concentration (LIC) using biosusceptometry, was a secondary objective. A total of 195 adult and paediatric patients received deferasirox (n = 132) or deferoxamine (n = 63). Adverse events most commonly associated with deferasirox were mild, including transient nausea, vomiting, diarrhoea, abdominal pain and skin rash. Abnormal laboratory studies with deferasirox were occasionally associated with mild non-progressive increases in serum creatinine and reversible elevations in liver function tests. Discontinuation rates from deferasirox (11·4%) and deferoxamine (11·1%) were similar. Over 1 year, similar dose-dependent LIC reductions were observed with deferasirox and deferoxamine. Once-daily oral deferasirox has acceptable tolerability and appears to have similar efficacy to deferoxamine in reducing iron burden in transfused patients with sickle cell disease.