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

Iron Overload Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01874405
• Other study ID #: DIPEDNA-2013-1
• Status: Completed
• Phase: N/A
• First received: May 26, 2013
• Last updated: June 6, 2013
• Start date: March 2003
• Est. completion date: October 2012

Study information

• Verified date: June 2013
• Source: Second University of Naples
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: Ethics Committee
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 80
• Est. completion date: October 2012
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years and older

Eligibility

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

Observational Model: Case-Only, Time Perspective: Retrospective

Related Conditions & MeSH terms

• Iron Overload

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Second University of Naples

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Primary	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	No
Secondary	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	No
Secondary	change in liver iron concentration		at least 1 year	No
Secondary	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	No