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

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

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

Brief Summary: 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.

Detailed Summary:
Sponsor: Second University of Naples

Current Primary Outcome:

  • cardiac T2* in patients treated with deferasirox [ Time Frame: at least 1 year ]
    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)
  • cardiac T2* in patients treated with deferasirox [ Time Frame: at least 1 year ]
    maintenance from baseline to end of study of cardiac T2* in not iron overloaded patients (cardiac T2* >20 ms at baseline)


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • cardiac function in patient undergoing deferasirox treatment [ Time Frame: at least 1 year ]
    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
  • change in liver iron concentration [ Time Frame: 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 [ Time Frame: at least 3 years ]
    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


Original Secondary Outcome: Same as current

Information By: Second University of Naples

Dates:
Date Received: May 26, 2013
Date Started: March 2003
Date Completion:
Last Updated: June 6, 2013
Last Verified: June 2013