Long-term safety and efficacy of deferasirox (Exjade®) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease

2011

Long-term safety and efficacy of deferasirox in sickle cell disease

Sample size: 185 publication 10 minutes Evidence: moderate

Author Information

Author(s): Vichinsky Elliott, Bernaudin Françoise, Forni Gian Luca, Gardner Renee, Hassell Kathryn, Heeney Matthew M, Inusa Baba, Kutlar Abdullah, Lane Peter, Mathias Liesl, Porter John, Tebbi Cameron, Wilson Felicia, Griffel Louis, Deng Wei, Giannone Vanessa, Coates Thomas

Primary Institution: Children's Hospital and Research Center at Oakland

Hypothesis

To evaluate the long-term safety and efficacy of deferasirox in transfusion-dependent patients with sickle cell disease.

Conclusion

Long-term treatment with deferasirox is safe and effective in reducing iron overload in patients with sickle cell disease.

Supporting Evidence

33.5% of patients completed the 5-year study.
Serum ferritin levels significantly decreased by −591 μg/l in patients with ≥4 years of deferasirox exposure.
Adverse events were predominantly mild-to-moderate and transient.

Takeaway

This study shows that a medicine called deferasirox can help kids and adults with sickle cell disease by safely reducing extra iron in their bodies over a long time.

Methodology

Patients with sickle cell disease and iron overload were treated with deferasirox for up to 5 years, with regular monitoring of serum ferritin levels and adverse events.