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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.
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| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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Adult and pediatric transfusion- dependent patients (male and female) with different underlying cronic anemias who received iron chelation therapy with deferasirox during the observational study period and underwent at least 2 cardiac MRI scans at the Pozzuoli site.
All consecutive patients visited at the participating sites starting from March 2003 to October 2012 will be entered in this observational study (chart review) provided all the inclusion an no exclusion criteria are met.
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| Name | Affiliation | Role |
|---|---|---|
| Silverio Perrotta, MD | University of Campania Luigi Vanvitelli | Principal Investigator |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25197009 | Derived | Casale M, Citarella S, Filosa A, De Michele E, Palmieri F, Ragozzino A, Amendola G, Pugliese U, Tartaglione I, Della Rocca F, Cinque P, Nobili B, Perrotta S. Endocrine function and bone disease during long-term chelation therapy with deferasirox in patients with beta-thalassemia major. Am J Hematol. 2014 Dec;89(12):1102-6. doi: 10.1002/ajh.23844. Epub 2014 Sep 26. |
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| ID | Term |
|---|---|
| D019190 | Iron Overload |
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000745 | Anemia, Hemolytic, Congenital |
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| 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 |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |