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

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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Key information

Conditions

Iron Overload Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Iron Metabolism Disorders Metabolic Diseases Nutritional and Metabolic Diseases Thalassemia

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

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)

Secondary outcomes

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

Sponsors and collaborators

Lead sponsor

University of Campania Luigi Vanvitelli

Other

Registry information

Official study title

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

Important dates

Study start: 2003
Primary completion: 2012
Study completion: 2012
First posted: Jun 11, 2013
Registry last updated: Jun 11, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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