Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease

Launched by CHILDREN'S HOSPITAL OF PHILADELPHIA · Feb 16, 2006

Keywords

ClinConnect Summary

Repeated red cell transfusions lead to transfusional iron overload because the body lacks an efficient mechanism to excrete excess iron. Without treatment, iron accumulates in the liver, heart and endocrine glands. Cardiac complications including arrhythmias and congestive heart failure are the most common cause of death from transfusional iron overload. New magnetic resonance imaging (MRI) T2\* techniques enable an estimation of cardiac iron loading, and allow patients at the highest risk of cardiac disease (those with T2\* \< 10 ms) to be identified. For over 30 years, deferoxamine has be...

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Eligibility criteria

• Inclusion Criteria:
• • Transfusional iron overload
• • Overt cardiac failure or significant arrhythmia, OR high risk of developing cardiac failure as determined by T2\* \< 10 ms by magnetic resonance imaging (MRI)
• • Signed consent form
• • Patient regularly followed at The Children's Hospital of Philadelphia
• • Unwillingness to participate in, or lack of suitability for, a clinical trial providing similar therapy
• Exclusion Criteria:
• • Previously treated with deferiprone and had severe adverse reactions necessitating discontinuation
• • Receiving other investigational drugs
• • Receiving other drugs known to cause neutropenia
• • Unexplained occurrences of neutropenia in past two years
• • Pregnant or breastfeeding; or want to become pregnant.
• • Sexually active but unwilling to use reliable birth control
• • Other conditions which, in the opinion of the investigator, would make patient unsuitable for enrollment