A 5 year observational study (registry) of children aged 2 to <6 years at enrollment with transfusional hemosiderosis treated with deferasirox (IRB# 25075)

Status: Active



  • Andrew S. Freiberg, M.D.



Study the long-term safety and effectiveness of Exjade (deferasirox, ICL670) a new, oral iron chelator in the youngest approved age group 2-<6.

The purpose of this study is to confirm that the drug deferasirox is safe when taken during 5 years by children aged between 2 and 6 years at the start of treatment.



  • 2-<6 years old
  • Eligible for treatment with deferasirox
  • Have transfusion-dependent anemias
  • Have iron overload as defined in the patients management guidelines
  • Beginning treatment with deferasirox for transfusional hemosiderosis at enrollment
  • Treatment with deferasirox <12 months prior to time of enrollment.

Exclusion Criteria    

  • Patients with non-transfusional hemosiderosis
  • Severe concomitant illnesses
  • Other involvement in a clinical trial.


Additional Study Details:

What is expected of me and my child?

There are no specific study procedures as the study is observational. Available data will be collected on any blood draws, blood transfusions, audiology, ophthalmology, weight, height, and SAEs.

Length of Study

  • 5 Years




  • Penn State Hershey Children's Hospital
  • HMC Pediatric and Medical Hematology Clinics



Jennifer Stokes, RN, BSN, CCRC




  • Hematology
  • Transfusional Hemosiderosis