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

NCT01874405 | Completed DMC

• 1 other identifier: DIPEDNA-2013-1
• Study Type: observational
• Target: 80
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Iron Overload

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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

• 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

Secondary Outcomes (3)

• cardiac function in patient undergoing deferasirox treatment

  at least 1 year

• 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

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• Non transfusion- dependent patients;
• Other chelation therapy than deferasirox;
• Absence of complete medical history as above specified

Sponsors & Collaborators

• University of Campania Luigi Vanvitelli: lead

Related Publications (1)

• 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.

  PMID: 25197009 DERIVED

MeSH Terms

Conditions

Iron Overload; Thalassemia

Condition Hierarchy (Ancestors)

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

Study Officials

• Silverio Perrotta, MD

  University of Campania Luigi Vanvitelli

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: May 26, 2013
• First Posted: June 11, 2013
• Study Start: March 1, 2003
• Primary Completion: October 1, 2012
• Study Completion: October 1, 2012
• Last Updated: June 11, 2013

Record last verified: 2013-06