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

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01874405

Enrollment

Registered

2013-06-11

Start date

2003-03-31

Completion date

2012-10-31

Last updated

2013-06-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Iron Overload

Keywords

Iron overload, thalassaemia, deferasirox, magnetic resonance, endocrine function

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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
cardiac T2* in patients treated with deferasirox	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)

Secondary

Measure	Time frame	Description
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

Outcome results