Clinical trial NCT01874405: Retrospective Evaluation of Adult and Pediatric Transfusion-dependent Patients Treated With Deferasirox Therapy
Official title: Effectiveness of Deferasirox Therapy on Comprehensive Management of Iron Overload in Adult and Pediatric Transfusion-dependent Patients: a Long Term Retrospective Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Iron Overload
Keywords: Iron overload
thalassaemia
deferasirox
magnetic resonance
endocrine function
Lead sponsor: University of Campania Luigi Vanvitelli
Sponsor class: OTHER
First posted: 2013-06-11
Enrollment: 80
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Adult and pediatric transfusion- dependent patients (male and female) with different underlying cronic anemias who received iron chelation therapy with deferasirox during the observational study period and underwent at least 2 cardiac MRI scans at the Pozzuoli site.

All consecutive patients visited at the participating sites starting from March 2003 to October 2012 will be entered in this observational study (chart review) provided all the inclusion an no exclusion criteria are met.

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.


Eligibility criteria: 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

Primary outcomes:
measure: cardiac T2* in patients treated with deferasirox
timeFrame: at least 1 year
measure: cardiac T2* in patients treated with deferasirox
timeFrame: at least 1 year
Secondary outcomes:
measure: cardiac function in patient undergoing deferasirox treatment
timeFrame: at least 1 year
measure: change in liver iron concentration
timeFrame: at least 1 year
measure: 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
timeFrame: at least 3 years
Other outcomes:
Locations:
Study officials:
affiliation: University of Campania Luigi Vanvitelli
name: Silverio Perrotta, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Casale M, Citarella S, Filosa A, De Michele E, Palmieri F, Ragozzino A, Amendola G, Pugliese U, Tartaglione I, Della Rocca F, Cinque P, Nobili B, Perrotta S. Endocrine function and bone disease during long-term chelation therapy with deferasirox in patients with beta-thalassemia major. Am J Hematol. 2014 Dec;89(12):1102-6. doi: 10.1002/ajh.23844. Epub 2014 Sep 26.