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

Completed

• Conditions: Iron Overload

• Registration Number: NCT01874405
• Lead Sponsor: University of Campania "Luigi Vanvitelli"

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 Description

Not available

Study Timeline

• Study Start: 2003-03
• Primary Completion: 2012-10
• Study Completion: 2012-10
• Study First Submitted: 2013-05-26
• Status Verified: 2013-06
• Study First Submitted QC: 2013-06-06
• Last Update Submitted: 2013-06-06
• Study First Posted: 2013-06-11
• Last Update Posted: 2013-06-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 80

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
cardiac T2* in patients treated with deferasirox	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)

Secondary Outcome Measures

Name	Time	Method
cardiac function in patient undergoing deferasirox treatment	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	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	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