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A five year observational study (registry) of children aged two to less than six years at enrollment with transfusional hemosiderosis treated with deferasirox

Dr. Elliott Vichinsky is the principal investigator of this study to collect safety and effectiveness data on pediatric patients taking Exjade for 5 years. This study is being done because there were only 52 pediatric patients aged 2 to less than 6 years enrolled in the studies that the FDA reviewed when Exjade was approved. Young children less than 6 years old typically receive a higher amount of blood transfused per body weight in comparison with adults or older children. More safety and effectiveness long-term data would be useful in this young patient population.

Who can participate?

    Inclusion Criteria
  • Patients who are chronically transfused
  • Patients who are between 2 and 6 years old
  • Patients who have been taking Exjade for less than a year or who are just beginning treatment with Exjade when they join the study
    Exclusion Criteria
  • Patients who have certain other serious diseases or conditions

What does the study involve?

Several times a year, a member of the study team will review the medical record and collect information about current drug treatment for iron overload, medical history (including illness and medicines or treatments), transfusion requirements, blood pressure, and growth and development. The results of any eye exams or hearing tests will be collected. Some lab results will also be collected. This is information that is usually recorded at visits to the hospital or clinic. Patients do not have to be seen at CHRCO to participate in this study. The study will reimburse $25 for each completed year.  There are no special tests or study visits required. The study does not provide Exjade.

For more information, please contact Jacqueline Madden at Children's Hospital & Research Center at Oakland, 510-428-3885, ext. 5745 or ask your clinical care provider.


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