Comparing Deferasirox and Deferoxamine for Iron Overload in Sickle Cell Disease
Author Information
Author(s): Vichinsky Elliott, Onyekwere Onyinye, Porter John, Swerdlow Paul, Eckman James, Lane Peter, Files Beatrice, Hassell Kathryn, Kelly Patrick, Wilson Felicia, Bernaudin Françoise, Forni Gian Luca, Okpala Iheanyi, Ressayre-Djaffer Catherine, Alberti Daniele, Holland Jaymes, Marks Peter, Fung Ellen, Fischer Roland, Mueller Brigitta U, Coates Thomas
Primary Institution: Children's Hospital and Research Center Oakland
Hypothesis
Is deferasirox as safe and effective as deferoxamine for treating transfusional iron overload in patients with sickle cell disease?
Conclusion
Deferasirox is well tolerated and has similar efficacy to deferoxamine in reducing iron burden in patients with sickle cell disease.
Supporting Evidence
- Adverse events were similar between the two treatments, with mild gastrointestinal symptoms being the most common.
- Both treatments resulted in significant reductions in liver iron concentration.
- Compliance with deferasirox was high due to its once-daily oral administration.
Takeaway
This study looked at two medicines to help people with sickle cell disease who have too much iron in their bodies. It found that both medicines work similarly well and are mostly safe to use.
Methodology
This was a randomised, open-label, phase II trial comparing the safety and efficacy of deferasirox and deferoxamine in patients with sickle cell disease and iron overload.
Potential Biases
Potential bias in patient selection and reporting of adverse events.
Limitations
The study duration was only one year, and the long-term safety of deferasirox in patients with abnormal renal function is unknown.
Participant Demographics
{"age":{"median":15,"range":"3-54"},"sex":{"female_percentage":59.0},"race":{"black_percentage":90.8,"caucasian_percentage":5.6}}
Statistical Information
P-Value
p<0.001 for deferasirox; p=0.022 for deferoxamine
Statistical Significance
p<0.05
Digital Object Identifier (DOI)
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