Retrospective Evaluation of Adult and Pediatric Transfusion-dependent Patients Treated With Deferasirox Therapy

Study Overview
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Brief Summary
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Iron overload is a leading cause of morbidity and mortality in transfusion-dependent patients. Deferasirox is the most promising iron chelator agent in several clinical scenarios. The investigators propose a retrospective study (chart review) to evaluate comprehensive iron overload management in transfusion-dependent patients treated with deferasirox for up to 5-10 years in a real clinical practice setting.

Official Title
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Effectiveness of Deferasirox Therapy on Comprehensive Management of Iron Overload in Adult and Pediatric Transfusion-dependent Patients: a Long Term Retrospective Study

Conditions
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Iron Overload

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Transfusion- dependent patients (> 2 years); Ongoing deferasirox therapy during the study period; ≥ 2 Magnetic Resonance scans (one at baseline and at least one post baseline - as per clinical need) during study period (this criteria is not mandatory for patients undergoing only the endocrine subanalysis and participating only to the cardiac analysis); Available medical history including relevant clinical and laboratory data (e.g serum ferritin, liver function tests, renal function tests, endocrine parameters ) at baseline before starting deferasirox treatment Exclusion Criteria: Non transfusion- dependent patients; Other chelation therapy than deferasirox; Absence of complete medical history as above specified

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| cardiac T2* in patients treated with deferasirox | change from baseline to end of study in cardiac T2*, as measured by Magnetic Resonance, in patients with iron overload (cardiac T2* <20 ms at baseline) | at least 1 year | 
| cardiac T2* in patients treated with deferasirox | maintenance from baseline to end of study of cardiac T2* in not iron overloaded patients (cardiac T2* >20 ms at baseline) | at least 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| cardiac function in patient undergoing deferasirox treatment | change in left and right ejection fraction, telediastolic and telesystolic volumes, stroke volumes, cardiac output, myocardial mass, measured by Cardiac Magnetic Resonance, from baseline to end of study | at least 1 year | 
| change in liver iron concentration |  | at least 1 year | 
| maintenance of normal endocrine function in patients without endocrine dysfunction and improvement in disease severity in patients affected by endocrine dysfunction from baseline to end of study | Thyroid function (TSH, free triiodothyronine and free thyroxine serum free T4 levels), pancreatic cell function (basal glycemia, glycated hemoglobin level), bone mineral density (z-score) will be evaluated by the closest assessment to baseline (first deferasirox exposure) and to the end of study | at least 3 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Iron overload, thalassaemia, deferasirox, magnetic resonance, endocrine function